RARE DISEASE
Central serous chorioretinopathy
Central serous chorioretinopathy
Central serous chorioretinopathy
Synonyms: CSCR
Synonyms: CSCR
Synonyms: CSCR
Drug discovery
2
drugs
With orphan designations
Overview
Central serous chorioretinopathy (CSCR) is a chorioretinal disorder characterized by serous detachment of the neurosensory retina due to fluid leakage from choroidal hyperpermeability. Primarily affecting adults aged 20-50, it disproportionately affects males (6:1 ratio) and is associated with glucocorticoid use, stress, type A personality, and obstructive sleep apnea [1][4][12]. While 50-80% of acute cases resolve spontaneously within 3-4 months, 30-50% develop chronic/recurrent disease requiring intervention [4][6][16].
Population
Burden
30-50% recurrence rate with 14-35% developing permanent RPE atrophy [4][6][12]
Annual incidence: 34/100,000 person-years, costing $18M/year in US working-age adults [7][9]
86% require no initial treatment, but 20% progress to chronic disease with reduced contrast sensitivity and metamorphopsia [7][18]
Categories: rare genetic diseases, rare ophthalmic disorders
Research Papers
1,237 drug discovery papers about Central serous chorioretinopathy, with 1 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,237 drug discovery papers about Central serous chorioretinopathy, with 1 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
categories:
Small molecules
small molecules
2026-08-06 | The "Tap-Drain" model of central serous chorioretinopathy: Mechanistic staging and a corrective action-preventative action-based framework for disease modification.
Central serous chorioretinopathy (CSCR) has traditionally been regarded as a self-limiting disorder and classified using duration- or phenotype-based frameworks. Accumulating clinical and imaging evidence, however, suggests that CSCR is a recurrent disorder in which choroidal haemodynamic abnormalities and retinal pigment epithelium (RPE) dysfunction interact to determine long-term structural and functional outcomes. We synthesize current advances in choroidal physiology and multimodal imaging to propose the Tap-Drain model, a physiology-based conceptual framework that reconceptualizes CSCR as a disorder of outer retinal fluid homeostasis. Within this framework, fluid entry ("tap") is proposed to arise predominantly from choroidal haemodynamic abnormalities, including vascular hyperpermeability and impaired venous drainage, whereas disease expression may also be influenced by regional variability in RPE functional reserve. Fluid clearance ("drain") is mediated by the RPE, and impaired RPE function may reduce its capacity to compensate for increased choroidal inflow, resulting in persistent or recurrent subretinal fluid (SRF). We further propose a mechanistic staging system and a Corrective Action-Preventive Action (CAPA) framework that aligns management with the predominant imbalance between fluid entry and clearance. By distinguishing tap-dominant, drain-stressed, and drain-failure phenotypes, the framework shifts treatment goals beyond episodic SRF resolution toward recurrence prevention, preservation of retinal structure, and maintenance of long-term visual function. Although biologically plausible and clinically intuitive, the Tap-Drain model remains a conceptual framework that requires prospective clinical validation before its prognostic and therapeutic implications can be considered established.
2026-07-30 | Evaluating the Efficacy and Safety of Topical Difluprednate in Patients with Chronic Central Serous Chorioretinopathy.
To evaluate the efficacy and safety of topical Difluprednate ophthalmic emulsion 0.05% in chronic Central Serous Chorioretinopathy (cCSCR) and compare its outcomes with oral eplerenone and observation. This pilot open-label randomised controlled trial included patients with cCSCR of ≥3 months' duration or relapsing cases with subfoveal or juxtafoveal leakage on angiography. After baseline assessment, patients were randomised into three groups: topical Difluprednate, oral Eplerenone, or observation. All participants were followed monthly for 3 months. Changes in best-corrected visual acuity, optical coherence tomography parameters, retinal sensitivity, and adverse events were documented. 53 adults (mean age 42.6 ± 9.5 years; 90.6% male) were enrolled. Complete resolution of subretinal fluid occurred in 90% (n=18) of the patients in the Difluprednate Group, 45% (n=9) in Eplerenone Group, and 23% (n=3) patients in the control arm (p < 0.001). The difluprednate group showed significantly greater reductions in subfoveal choroidal thickness (SFCT) and improvements in 3-mm microperimetry thresholds compared with the observation group (p = 0.012 and p = 0.022, respectively). It also demonstrated the largest decrease in central macular thickness, although this was not statistically significant (p = 0.19). Ocular hypertension developed in 30% of difluprednate-treated eyes and was controlled with topical antiglaucoma medication. Topical difluprednate produced superior anatomical and functional improvements in SRF resolution, SFCT reduction, and retinal sensitivity within 3-mm when compared with oral eplerenone and observation. Thus, with a manageable safety profile, it may serve as an effective therapeutic option for cCSCR.
2026-07-28 | Photodynamic Therapy in Central Serous Chorioretinopathy: Treatment Timing, Anatomical Response, and Recurrence in a Large Real-World Cohort.
Central serous chorioretinopathy (CSC) is characterized by pathological accumulation of subretinal fluid (SRF), leading to visual impairment. Photodynamic therapy (PDT) is an established treatment for chronic or persistent CSC. This study aimed to identify predictors of treatment response in a large cohort of patients with CSC treated with PDT. In this retrospective, single-center study, clinical characteristics and treatment outcomes of patients with CSC undergoing PDT were systematically analyzed. Central retinal thickness (CRT) and visual acuity were assessed before and after treatment. The interval between initial diagnosis and PDT was evaluated for its impact on therapeutic response. A total of 115 patients (90 males, 25 females; mean age 49.95 ± 10.15 years) were included. The mean interval from diagnosis to PDT was 3.79 ± 3.53 years. CRT decreased significantly following PDT (p < 0.001), with complete SRF resolution in 51.3% of eyes. Mean visual acuity improved by -0.02 ± 0.22 logMAR (p = 0.0047). Recurrence occurred in 30% of eyes after a mean of 1.1 ± 1.1 years. A significant negative correlation was observed between time to PDT and CRT reduction (r = -0.286, p = 0.001) but not with visual acuity (p = 0.76). Repeat PDT, performed in 16 eyes, resulted in further significant CRT reduction (p = 0.008). PDT is effective in reducing SRF in CSC. Delayed treatment was associated with diminished anatomical response. Repeat PDT may provide additional benefit in recurrent disease.
2026-07-28 | Functional and Structural Outcomes of Photodynamic Therapy (PDT) With or Without Eplerenone for Central Serous Chorioretinopathy.
Background/Objectives: To evaluate whether the combination of oral eplerenone and half-dose full-fluence photodynamic therapy (HD-FF PDT) provides greater efficacy than HD-FF PDT alone in persistent central serous chorioretinopathy (CSCR). Methods: This monocentric, retrospective, observational study included patients with persistent (>6 months) simple or complex CSCR who had previously undergone either half-dose full-fluence photodynamic therapy (HD-FF PDT) alone or HD-FF PDT combined with oral eplerenone as part of routine clinical practice between September 2024 and March 2025. Functional and morphological data collected at baseline and at 1, 3, and 6 months after treatment were retrospectively reviewed. An artificial intelligence-based algorithm was used to analyze OCT scans, quantifying subretinal and intraretinal fluid volumes (SRFV, IRFV) and assessing ellipsoid zone and external limiting membrane integrity, hyperreflective foci, subfoveal choroidal thickness (SCT), and central macular thickness (CMT). Results: Fifty patients (53 eyes; mean age 52 years) were included, with no significant baseline differences between groups. Best-corrected visual acuity improved more significantly in Group B at 1 and 6 months (p = 0.032 and p = 0.009, respectively). At 6 months, subretinal fluid volume (SRFV), quantified by AI-based OCT analysis, was significantly lower in the combined therapy group (p = 0.014). An AI-defined complete resolution of subretinal fluid (SRFV < 0.010 mm3) was achieved more frequently in Group B than in Group A (77% vs. 22%, p = 0.001). Conclusions: Although HD-FF PDT remains the standard treatment for persistent CSCR, adjunctive therapy with the mineralocorticoid receptor antagonist eplerenone may enhance subretinal fluid reabsorption and improve mid-term anatomical and functional outcomes.
2026-07-27 | Optical Coherence Tomography Angiography-Guided Laser Management of Neovascular Central Serous Chorioretinopathy.
To analyze the efficacy and safety of optical coherence tomography angiography (OCTA)-based navigated laser treatment in central serous chorioretinopathy (CSCR) complicated by choroidal neovascularization (CNV). This study included consecutive patients with neovascular non-resolving CSCR who received en face OCT-based navigated laser treatment without fluorescein angiography or were observed untreated for at least six months. In all treated cases the area of the double-layer sign with flow signal displayed with structural en face OCT image was treated with microsecond pulsing laser (up to three sessions), direct continuous wave focal laser photocoagulation, or their consecutive combination. Main outcome measure was complete resolution of subretinal fluid. Sixty-seven eyes of 67 patients (28 males, 41.8%) with a mean age of 57.5 ± 7.7 years were included in the treatment group. Thirty eyes of 30 patients (14 males (46.7%), mean age 58.1 ± 6.9 years) were included in the control group. Complete resolution of subretinal fluid was achieved in 32 eyes (47.8%) in the treatment group compared with none in the control group (p < 0.001). The odds ratio for the success of the laser treatment in the presence of fovea-involving CNV was 0.21 (95% CI 0.073 to 0.583). After excluding eyes with CNV involving the foveal center, the success rate achieved was 65.0%. No adverse effects in the treatment group were observed except two cases of CNV growth after focal laser photocoagulation. OCTA-guided laser treatment is a viable and safe option in the management of CSCR complicated by CNV, mostly effective in cases with extrafoveal CNV.
antibodies
2026-07-01 | Central Serous Chorioretinopathy: Current Concepts in Pathogenesis, Multimodal Imaging, and Management
Central serous chorioretinopathy (CSC) is a retinal disorder characterised by serous detachment of the neurosensory retina. It results from dysfunction of the retinal pigment epithelium and derangements in choroidal circulation. Once regarded as a benign and self-limiting macular disease of young men, CSC is now recognised as part of a broader pachychoroid spectrum in which choroidal vascular congestion, venous overload, and hyperpermeability play crucial roles. Multimodal imaging, including optical coherence tomography (OCT), fundus autofluorescence, indocyanine green angiography, and OCT-angiography, have significantly altered the understanding of this condition and allowed early identification of chronicity and complications such as choroidal neovascularisation. Management strategies continue to evolve. Although the availability of photodynamic therapy may be limited in some settings, micropulse laser, focal thermal laser, anti-vascular endothelial growth factor therapy, and selected systemic medications continue to have important roles in specific clinical scenarios. This narrative review provides an updated synthesis of current knowledge regarding CSC, emphasising its pathogenesis, clinical presentation, imaging findings, complications, and therapeutic approaches.
2026-06-20 | Research advances in claudin-1 in the eye.
Claudin-1, a transmembrane protein integral to tight junctions, is indispensable for paracellular barrier formation and the establishment and maintenance of cellular polarity. In the eye, claudin-1 exhibits tissue-specific expression in critical structures such as the cornea and retina, where it contributes fundamentally to corneal transparency, blood-retinal barrier integrity, and retinal homeostasis. This review comprehensively synthesizes current knowledge on the spatial distribution and physiological roles of claudin-1 across ocular tissues. We critically evaluate recent mechanistic and translational advances implicating claudin-1 dysregulation in a spectrum of ocular pathologies: anterior segment disorders (dry eye disease, pterygium, infectious keratitis, and gelatinous drop-like corneal dystrophy), retinal diseases (diabetic retinopathy, age-related macular degeneration, central serous chorioretinopathy, and retinoblastoma), and other conditions (uveitis, glaucoma, post-cataract macular edema, and cataract). Finally, we assess the emerging therapeutic potential of claudin-1-both as a molecular target for barrier modulation and as a facilitator of paracellular drug delivery in ophthalmology.
2026-06-02 | Evaluating the Correlation Between Optical Coherence Tomography Angiography-Derived Parameters and Serum Vascular Endothelial Growth Factor-A (VEGF-A) Levels in Central Serous Chorioretinopathy.
Release of vascular endothelial growth factor-A (VEGF-A) is one of the mechanisms involved in the pathogenesis of central serous chorioretinopathy (CSCR). We intended to evaluate the correlation between optical coherence tomography angiography (OCTA) parameters and serum VEGF-A levels in CSCR. Our prospective interventional study included 31 patients diagnosed with CSCR. The subjects had a mean age of 39.23±6.9 (26-53) years. They were examined for visual acuity (VA) and tested for color perception and contrast discrimination. OCT was conducted to examine central macular thickness (CMT) and subfoveal choroidal thickness (SFCT), and to detect neurosensory detachment (NSD), pigment epithelial detachment (PED), and other abnormalities. The OCTA measured foveal avascular zone (FAZ) and vessel density (VD) across various retino-choroidal layers. The serum VEGF-A levels were measured using commercially available enzyme-linked immunosorbent assay (ELISA) kits. The eyes were treated with a single intravitreal injection of 0.3 mg/0.05 ml ranibizumab biosimilar (RbB). After one month, the ocular parameters were rechecked, and serum VEGF-A levels were remeasured. We assessed the correlation between ocular parameters and VEGF-A levels in serum samples at both instances. At baseline, the mean value of the best corrected visual acuity (BCVA) in logarithm of the minimum angle of resolution (LogMAR) was 0.81±0.43. The CMT measured 286.81±117.67 microns, SFCT 194.32±62.76 microns, and the serum VEGF-A level was 351.64 (IQR 243.57-478.71) pg/ml. Before treatment, circulating VEGF-A levels correlated positively with the LogMAR value of BCVA (Rho=0.121), CMT (Rho=0.282), SFCT (Rho=0.006), NSD height (Rho=0.100) and width (Rho=0.019), and PED number (Rho=0.016), height (Rho=0.246) and width (Rho=0.066). The serum VEGF-A showed a weak positive correlation (p>0.05) with most OCTA features, whereas it correlated negatively with FAZ area (Rho=-0.136) and perimeter (Rho=-0.128). A month after intravitreal injection, LogMAR BCVA improved to 0.42±0.42, CMT measured 256.65±109.57 microns, and SFCT was 209.84±41.53 microns. The median serum VEGF-A level was 336.31 (IQR 259.455-500.105) pg/ml. Serum VEGF-A showed weak positive correlation with LogMAR value of BCVA (Rho=0.046), CMT (Rho=0.063), SFCT (Rho=0.094), NSD number (Rho=0.290), height (Rho=0.269) and width (Rho=0.289), and PED number (Rho=0.306), height (Rho=0.204) and width (Rho=0.124). The serum VEGF-A showed negative weak correlations (p>0.05) with OCTA features, including FAZ metrics --area (Rho=-0.156), perimeter (Rho=-0.114), and circularity (Rho=-0.335), as well as VD in various retino-choroidal layers: superficial capillary plexus (Rho=-0.282); deep capillary plexus (Rho=-0.225), outer retinal chorio-capillaries (Rho=-0.295), chorio-capillaries (Rho=-0.312) and choroid (Rho=-0.274). Higher serum VEGF-A was associated with worse VA and increased CMT, SFCT, NSD height and width, and PED number, height, and width. An increase in VEGF-A was related to reduced FAZ area and perimeter. At baseline, higher levels of VEGF-A were accompanied by an increase in VD in all layers except the outer retina and choroid. After treatment with intravitreal RbB, a similar pattern was observed; however, increased VEGF-A was found to be coupled with decreased VD in all retino-choroidal layers, except the outer retina.
2026-05-27 | Effects of Selective Retina Therapy on Central Serous Chorioretinopathy with Serous Pigment Epithelial Detachments.
Background/Objective: This study's aim is to evaluate the anatomical and functional effects of selective retina therapy (SRT) in patients with central serous chorioretinopathy (CSC) accompanied by serous pigment epithelial detachment (PED). Methods: This retrospective study included 32 eyes from 32 patients with CSC and serous PED treated with SRT. Pulse energy and micropulse number were adjusted based on test spot visibility on fundus photographs. Best-corrected visual acuity (BCVA; logMAR), central foveal thickness (CFT), subretinal fluid (SRF) height, PED height, and subfoveal choroidal thickness were assessed at baseline and at 1, 2, and 3 months post-treatment. Retinal sensitivity was evaluated using microperimetry at baseline and 3 months. Results: At 3 months after SRT, complete SRF resolution was achieved in 78.1% of eyes (25/32). Mean BCVA improved significantly from 0.29 ± 0.30 logMAR at baseline to 0.20 ± 0.29 logMAR (p = 0.006). Mean CFT decreased from 284.7 ± 91.3 µm to 165.7 ± 94.8 µm (p < 0.001). Mean SRF height decreased from 150.5 ± 74.6 µm to 20.9 ± 48.3 µm (p < 0.001), and mean PED height decreased from 101.7 ± 96.9 µm to 33.3 ± 37.6 µm (p < 0.001). Retinal sensitivity showed a non-significant improvement at 3 months (p = 0.108). Reduction in PED height was moderately correlated with reduction in SRF height (r = 0.446, p = 0.011). Conclusions: SRT was associated with reductions in PED and SRF in CSC. These findings should be interpreted cautiously given the absence of a control group and the potential for spontaneous changes in SRF and PED.
2026-05-27 | Long-term analysis of intravitreal brolucizumab in recalcitrant cases of chronic central serous retinopathy complicated by neovascularization (pachychoroid neovasculopathy).
To assess the long-term safety and efficacy of intravitreal brolucizumab (Pagenax®) in eyes with chronic central serous chorioretinopathy (CSC) complicated by neovascularization (pachychoroid neovasculopathy) refractory to prior therapies. In this single-center retrospective case series (January 2021-July 2025), 30 eyes of 25 patients with recalcitrant and refractory cases of chronic CSC with secondary neovascularization were treated off-label with pro-re-nata intravitreal brolucizumab. Best-corrected visual acuity (BCVA), central subfield thickness (CST), and pigment epithelial detachment (PED) height were measured at baseline and 6 months, with extended outcomes in nine eyes assessed at 1 and 2 years. Adverse events were recorded at each visit. At 6 months, the mean CST decreased significantly from 328 ± 222 µm to 172 ± 79 µm ( P < 0.001), and the PED height declined from 262 ± 192 µm to 155 ± 123 µm ( P = 0.027). BCVA improved or remained stable in 90% of eyes, with no significant change in mean logMAR (0.38 to 0.33, P = 0.314). In the extended cohort, CST reductions persisted at 1 year (412 ± 241 µm to 271 ± 239 µm, P = 0.009) and 2 years (412 ± 241 µm to 164 ± 42 µm, P = 0.004), while BCVA stayed stable. A weak correlation suggested more injections might be linked to slightly poorer BCVA, possibly due to resistant cases requiring more treatment. No adverse events were noted. Intravitreal brolucizumab administered PRN effectively resolves persistent fluid and reduces PED in chronic CSC with PNV refractory to prior treatments, while maintaining stable visual acuity and demonstrating a favorable safety profile through 2 years of follow-up.
gene therapies
2022-08-10 | An Insertion Variant in CRH Confers an Increased Risk of Central Serous Chorioretinopathy.
To identify a novel corticotropin-releasing hormone (CRH) gene variant relevant in patients with central serous chorioretinopathy (CSC). We performed a genetic study of CSC in families and sporadic cases with controls. Using whole-exome sequencing and linkage analysis, we identified a heterozygous insertion variant, Gln52insPro, in the CRH gene that cosegregated in two Chinese families with CSC. This variant was evaluated among an additional 1307 patients with CSC and 1438 ethnicity-matched control individuals from three independent Chinese cohorts. The CRH variant was strongly associated with CSC in these cohorts of Chinese patients (Pmeta = 1.24 × 10-11; odds ratio, 3.01; 95% confidence interval, 2.15-4.21). The risk variant Gln52insPro decreased CRH gene expression. Our results implicate the hypothalamic-pituitary-adrenal stress response system in the pathogenesis of CSC and provide a novel rationale for therapeutic intervention.
2015-11-01 | Selective Retina Therapy
The safety and effectiveness of Selective Retina Therapy as a procedure that induces the regeneration of the retinal pigmented epithelial cells by selectively destroying only the retinal pigmented epithelial cell in patients with retinal diseases were assessed. Assessment was made on the Selective Retina Therapy by extending the range of patients to be subjected to this technology by including macular edema and central serous chorioretinopathy patients. Intervention procedures included a broad range of technologies that selectively damages the retinal pigments and all devices without limitation were selected in assessment of the safety and effectiveness of this technology. For information search, 8 domestic databases including Korea Med and overseas databases including Ovid-Medline, Ovid-Embase and Cochrane Library were used. A total of 55 relevant literatures were searched through the use of search words related to ‘selective retina therapy’. As the result, a total of 7 literatures were included in the final assessment by applying the criteria for selection and exclusion to the 28 literatures after having excluded 27 overlappingly searched literatures. Each of the stages from literature search to application of selection standards and extraction of data were carried out independently by 2 assessors under the deliberation by the Sub-committee. Although there is no safety problem with the Selective Retina Therapy when implemented on central serous chorioretinopathy patient, there is no literature that made report on the key effectiveness variable of the Therapy,making the assessment of the effectiveness of this technology difficulty. Therefore, this technology was assessed to be at a stage that requires further researches (Recommendation rating of D, Classification as technology in research stage i).
2012-01-17 | Suprachoroidal Electrotransfer: A Nonviral Gene Delivery Method to Transfect the Choroid and the Retina Without Detaching the Retina
Photoreceptors and retinal pigment epithelial cells (RPE) targeting remains challenging in ocular gene therapy. Viral gene transfer, the only method having reached clinical evaluation, still raises safety concerns when administered via subretinal injections. We have developed a novel transfection method in the adult rat, called suprachoroidal electrotransfer (ET), combining the administration of nonviral plasmid DNA into the suprachoroidal space with the application of an electrical field. Optimization of injection, electrical parameters and external electrodes geometry using a reporter plasmid, resulted in a large area of transfected tissues. Not only choroidal cells but also RPE, and potentially photoreceptors, were efficiently transduced for at least a month when using a cytomegalovirus (CMV) promoter. No ocular complications were recorded by angiographic, electroretinographic, and histological analyses, demonstrating that under selected conditions the procedure is devoid of side effects on the retina or the vasculature integrity. Moreover, a significant inhibition of laser induced-choroidal neovascularization (CNV) was achieved 15 days after transfection of a soluble vascular endothelial growth factor receptor-1 (sFlt-1)-encoding plasmid. This is the first nonviral gene transfer technique that is efficient for RPE targeting without inducing retinal detachment. This novel minimally invasive nonviral gene therapy method may open new prospects for human retinal therapies.
cell therapies
2026-08-11 | Role of Circulating Immune Cells in Mediating the Effect of Gut Microbiota on Central Serous Chorioretinopathy: A Mendelian Randomization and Mediation Analysis.
Recent observational studies have revealed gut microbiota influences the development and progression of several retinal diseases. However, the causal relationship between gut microbiota and central serous chorioretinopathy (CSCR) are still not understood. The data of gut microbiota and circulating immune cell traits were identifed from large‑scale genome‑wide association studies (GWAS) summary data, and the data of CSCR come from FinnGen database.Mendelian randomization (MR) was used to investigate the causal relationships between gut microbiota, circulating immune cells, and CSCR. Mendelian randomization methods included inverse variance weighting and Bayesian weighting. Cochran's Q, Egger intercept, and MR-PRESSO were used to evaluate heterogeneity, genetic pleiotropy, and horizontal pleiotropy. Sensitivity was evaluated using leave-one-out method.Finally, we explored whether immune cell act as a mediating factor in the pathway from gut microbiota to CSCR. 12 gut microbiota traits were associated with increased genetic risk of CSCR, 2 were associated with decreased genetic risk of CSCR.Mediated mendelian randomization analysis identified 10 pathways through which gut microbiota affects the genetic prediction of CSCR through circulating immune cell mediation, with Demequinaceae via IgD-CD24-% lymphocyte (7.0%) and CD24 on sw mem (3.5%), Demequina via IgD-CD24-% lymphocyte (9.6%), GCA-900066575 sp900066385 via CD4 on CD28 + CD4+ (9.7%), Saccharofermentanaceae via CD45 on HLA DR+T cells (4.7%), CD80 on plasmacytoid DC (5.0%), and CD80 on CD62L + plasmacytoid DC (5.9%), UBA737 via HLA DR+CD8br AC (4.3%), CAG-1000 sp000434555 via Resting Treg% CD4 Treg (3.3%) affecting the genetic risk of CSCR. Our mediated mendelian randomization analysis provides genetic evidence suggesting that circulating immune cells may mediate the causal relationship between gut microbiota and CSCR. The identified associations and mediation effects offer new insights into potential therapeutic avenues for CSCR.
2022-04-21 | Exploring the choroidal vascular labyrinth and its molecular and structural roles in health and disease.
The choroid is a key player in maintaining ocular homeostasis and plays a role in a variety of chorioretinal diseases, many of which are poorly understood. Recent advances in the field of single-cell RNA sequencing have yielded valuable insights into the properties of choroidal endothelial cells (CECs). Here, we review the role of the choroid in various physiological and pathophysiological mechanisms, focusing on the role of CECs. We also discuss new insights regarding the phenotypic properties of CECs, CEC subpopulations, and the value of measuring transcriptomics in primary CEC cultures derived from post-mortem eyes. In addition, we discuss key phenotypic, structural, and functional differences that distinguish CECs from other endothelial cells such as retinal vascular endothelial cells. Understanding the specific clinical and molecular properties of the choroid will shed new light on the pathogenesis of the broad clinical range of chorioretinal diseases such as age-related macular degeneration, central serous chorioretinopathy and other diseases within the pachychoroid spectrum, uveitis, and diabetic choroidopathy. Although our knowledge is still relatively limited with respect to the clinical features and molecular pathways that underlie these chorioretinal diseases, we summarise new approaches and discuss future directions for gaining new insights into these sight-threatening diseases and highlight new therapeutic strategies such as pluripotent stem cell‒based technologies and gene therapy.
small molecules
2026-08-06 | The "Tap-Drain" model of central serous chorioretinopathy: Mechanistic staging and a corrective action-preventative action-based framework for disease modification.
Central serous chorioretinopathy (CSCR) has traditionally been regarded as a self-limiting disorder and classified using duration- or phenotype-based frameworks. Accumulating clinical and imaging evidence, however, suggests that CSCR is a recurrent disorder in which choroidal haemodynamic abnormalities and retinal pigment epithelium (RPE) dysfunction interact to determine long-term structural and functional outcomes. We synthesize current advances in choroidal physiology and multimodal imaging to propose the Tap-Drain model, a physiology-based conceptual framework that reconceptualizes CSCR as a disorder of outer retinal fluid homeostasis. Within this framework, fluid entry ("tap") is proposed to arise predominantly from choroidal haemodynamic abnormalities, including vascular hyperpermeability and impaired venous drainage, whereas disease expression may also be influenced by regional variability in RPE functional reserve. Fluid clearance ("drain") is mediated by the RPE, and impaired RPE function may reduce its capacity to compensate for increased choroidal inflow, resulting in persistent or recurrent subretinal fluid (SRF). We further propose a mechanistic staging system and a Corrective Action-Preventive Action (CAPA) framework that aligns management with the predominant imbalance between fluid entry and clearance. By distinguishing tap-dominant, drain-stressed, and drain-failure phenotypes, the framework shifts treatment goals beyond episodic SRF resolution toward recurrence prevention, preservation of retinal structure, and maintenance of long-term visual function. Although biologically plausible and clinically intuitive, the Tap-Drain model remains a conceptual framework that requires prospective clinical validation before its prognostic and therapeutic implications can be considered established.
2026-07-30 | Evaluating the Efficacy and Safety of Topical Difluprednate in Patients with Chronic Central Serous Chorioretinopathy.
To evaluate the efficacy and safety of topical Difluprednate ophthalmic emulsion 0.05% in chronic Central Serous Chorioretinopathy (cCSCR) and compare its outcomes with oral eplerenone and observation. This pilot open-label randomised controlled trial included patients with cCSCR of ≥3 months' duration or relapsing cases with subfoveal or juxtafoveal leakage on angiography. After baseline assessment, patients were randomised into three groups: topical Difluprednate, oral Eplerenone, or observation. All participants were followed monthly for 3 months. Changes in best-corrected visual acuity, optical coherence tomography parameters, retinal sensitivity, and adverse events were documented. 53 adults (mean age 42.6 ± 9.5 years; 90.6% male) were enrolled. Complete resolution of subretinal fluid occurred in 90% (n=18) of the patients in the Difluprednate Group, 45% (n=9) in Eplerenone Group, and 23% (n=3) patients in the control arm (p < 0.001). The difluprednate group showed significantly greater reductions in subfoveal choroidal thickness (SFCT) and improvements in 3-mm microperimetry thresholds compared with the observation group (p = 0.012 and p = 0.022, respectively). It also demonstrated the largest decrease in central macular thickness, although this was not statistically significant (p = 0.19). Ocular hypertension developed in 30% of difluprednate-treated eyes and was controlled with topical antiglaucoma medication. Topical difluprednate produced superior anatomical and functional improvements in SRF resolution, SFCT reduction, and retinal sensitivity within 3-mm when compared with oral eplerenone and observation. Thus, with a manageable safety profile, it may serve as an effective therapeutic option for cCSCR.
2026-07-28 | Photodynamic Therapy in Central Serous Chorioretinopathy: Treatment Timing, Anatomical Response, and Recurrence in a Large Real-World Cohort.
Central serous chorioretinopathy (CSC) is characterized by pathological accumulation of subretinal fluid (SRF), leading to visual impairment. Photodynamic therapy (PDT) is an established treatment for chronic or persistent CSC. This study aimed to identify predictors of treatment response in a large cohort of patients with CSC treated with PDT. In this retrospective, single-center study, clinical characteristics and treatment outcomes of patients with CSC undergoing PDT were systematically analyzed. Central retinal thickness (CRT) and visual acuity were assessed before and after treatment. The interval between initial diagnosis and PDT was evaluated for its impact on therapeutic response. A total of 115 patients (90 males, 25 females; mean age 49.95 ± 10.15 years) were included. The mean interval from diagnosis to PDT was 3.79 ± 3.53 years. CRT decreased significantly following PDT (p < 0.001), with complete SRF resolution in 51.3% of eyes. Mean visual acuity improved by -0.02 ± 0.22 logMAR (p = 0.0047). Recurrence occurred in 30% of eyes after a mean of 1.1 ± 1.1 years. A significant negative correlation was observed between time to PDT and CRT reduction (r = -0.286, p = 0.001) but not with visual acuity (p = 0.76). Repeat PDT, performed in 16 eyes, resulted in further significant CRT reduction (p = 0.008). PDT is effective in reducing SRF in CSC. Delayed treatment was associated with diminished anatomical response. Repeat PDT may provide additional benefit in recurrent disease.
2026-07-28 | Functional and Structural Outcomes of Photodynamic Therapy (PDT) With or Without Eplerenone for Central Serous Chorioretinopathy.
Background/Objectives: To evaluate whether the combination of oral eplerenone and half-dose full-fluence photodynamic therapy (HD-FF PDT) provides greater efficacy than HD-FF PDT alone in persistent central serous chorioretinopathy (CSCR). Methods: This monocentric, retrospective, observational study included patients with persistent (>6 months) simple or complex CSCR who had previously undergone either half-dose full-fluence photodynamic therapy (HD-FF PDT) alone or HD-FF PDT combined with oral eplerenone as part of routine clinical practice between September 2024 and March 2025. Functional and morphological data collected at baseline and at 1, 3, and 6 months after treatment were retrospectively reviewed. An artificial intelligence-based algorithm was used to analyze OCT scans, quantifying subretinal and intraretinal fluid volumes (SRFV, IRFV) and assessing ellipsoid zone and external limiting membrane integrity, hyperreflective foci, subfoveal choroidal thickness (SCT), and central macular thickness (CMT). Results: Fifty patients (53 eyes; mean age 52 years) were included, with no significant baseline differences between groups. Best-corrected visual acuity improved more significantly in Group B at 1 and 6 months (p = 0.032 and p = 0.009, respectively). At 6 months, subretinal fluid volume (SRFV), quantified by AI-based OCT analysis, was significantly lower in the combined therapy group (p = 0.014). An AI-defined complete resolution of subretinal fluid (SRFV < 0.010 mm3) was achieved more frequently in Group B than in Group A (77% vs. 22%, p = 0.001). Conclusions: Although HD-FF PDT remains the standard treatment for persistent CSCR, adjunctive therapy with the mineralocorticoid receptor antagonist eplerenone may enhance subretinal fluid reabsorption and improve mid-term anatomical and functional outcomes.
2026-07-27 | Optical Coherence Tomography Angiography-Guided Laser Management of Neovascular Central Serous Chorioretinopathy.
To analyze the efficacy and safety of optical coherence tomography angiography (OCTA)-based navigated laser treatment in central serous chorioretinopathy (CSCR) complicated by choroidal neovascularization (CNV). This study included consecutive patients with neovascular non-resolving CSCR who received en face OCT-based navigated laser treatment without fluorescein angiography or were observed untreated for at least six months. In all treated cases the area of the double-layer sign with flow signal displayed with structural en face OCT image was treated with microsecond pulsing laser (up to three sessions), direct continuous wave focal laser photocoagulation, or their consecutive combination. Main outcome measure was complete resolution of subretinal fluid. Sixty-seven eyes of 67 patients (28 males, 41.8%) with a mean age of 57.5 ± 7.7 years were included in the treatment group. Thirty eyes of 30 patients (14 males (46.7%), mean age 58.1 ± 6.9 years) were included in the control group. Complete resolution of subretinal fluid was achieved in 32 eyes (47.8%) in the treatment group compared with none in the control group (p < 0.001). The odds ratio for the success of the laser treatment in the presence of fovea-involving CNV was 0.21 (95% CI 0.073 to 0.583). After excluding eyes with CNV involving the foveal center, the success rate achieved was 65.0%. No adverse effects in the treatment group were observed except two cases of CNV growth after focal laser photocoagulation. OCTA-guided laser treatment is a viable and safe option in the management of CSCR complicated by CNV, mostly effective in cases with extrafoveal CNV.
antibodies
2026-07-01 | Central Serous Chorioretinopathy: Current Concepts in Pathogenesis, Multimodal Imaging, and Management
Central serous chorioretinopathy (CSC) is a retinal disorder characterised by serous detachment of the neurosensory retina. It results from dysfunction of the retinal pigment epithelium and derangements in choroidal circulation. Once regarded as a benign and self-limiting macular disease of young men, CSC is now recognised as part of a broader pachychoroid spectrum in which choroidal vascular congestion, venous overload, and hyperpermeability play crucial roles. Multimodal imaging, including optical coherence tomography (OCT), fundus autofluorescence, indocyanine green angiography, and OCT-angiography, have significantly altered the understanding of this condition and allowed early identification of chronicity and complications such as choroidal neovascularisation. Management strategies continue to evolve. Although the availability of photodynamic therapy may be limited in some settings, micropulse laser, focal thermal laser, anti-vascular endothelial growth factor therapy, and selected systemic medications continue to have important roles in specific clinical scenarios. This narrative review provides an updated synthesis of current knowledge regarding CSC, emphasising its pathogenesis, clinical presentation, imaging findings, complications, and therapeutic approaches.
2026-06-20 | Research advances in claudin-1 in the eye.
Claudin-1, a transmembrane protein integral to tight junctions, is indispensable for paracellular barrier formation and the establishment and maintenance of cellular polarity. In the eye, claudin-1 exhibits tissue-specific expression in critical structures such as the cornea and retina, where it contributes fundamentally to corneal transparency, blood-retinal barrier integrity, and retinal homeostasis. This review comprehensively synthesizes current knowledge on the spatial distribution and physiological roles of claudin-1 across ocular tissues. We critically evaluate recent mechanistic and translational advances implicating claudin-1 dysregulation in a spectrum of ocular pathologies: anterior segment disorders (dry eye disease, pterygium, infectious keratitis, and gelatinous drop-like corneal dystrophy), retinal diseases (diabetic retinopathy, age-related macular degeneration, central serous chorioretinopathy, and retinoblastoma), and other conditions (uveitis, glaucoma, post-cataract macular edema, and cataract). Finally, we assess the emerging therapeutic potential of claudin-1-both as a molecular target for barrier modulation and as a facilitator of paracellular drug delivery in ophthalmology.
2026-06-02 | Evaluating the Correlation Between Optical Coherence Tomography Angiography-Derived Parameters and Serum Vascular Endothelial Growth Factor-A (VEGF-A) Levels in Central Serous Chorioretinopathy.
Release of vascular endothelial growth factor-A (VEGF-A) is one of the mechanisms involved in the pathogenesis of central serous chorioretinopathy (CSCR). We intended to evaluate the correlation between optical coherence tomography angiography (OCTA) parameters and serum VEGF-A levels in CSCR. Our prospective interventional study included 31 patients diagnosed with CSCR. The subjects had a mean age of 39.23±6.9 (26-53) years. They were examined for visual acuity (VA) and tested for color perception and contrast discrimination. OCT was conducted to examine central macular thickness (CMT) and subfoveal choroidal thickness (SFCT), and to detect neurosensory detachment (NSD), pigment epithelial detachment (PED), and other abnormalities. The OCTA measured foveal avascular zone (FAZ) and vessel density (VD) across various retino-choroidal layers. The serum VEGF-A levels were measured using commercially available enzyme-linked immunosorbent assay (ELISA) kits. The eyes were treated with a single intravitreal injection of 0.3 mg/0.05 ml ranibizumab biosimilar (RbB). After one month, the ocular parameters were rechecked, and serum VEGF-A levels were remeasured. We assessed the correlation between ocular parameters and VEGF-A levels in serum samples at both instances. At baseline, the mean value of the best corrected visual acuity (BCVA) in logarithm of the minimum angle of resolution (LogMAR) was 0.81±0.43. The CMT measured 286.81±117.67 microns, SFCT 194.32±62.76 microns, and the serum VEGF-A level was 351.64 (IQR 243.57-478.71) pg/ml. Before treatment, circulating VEGF-A levels correlated positively with the LogMAR value of BCVA (Rho=0.121), CMT (Rho=0.282), SFCT (Rho=0.006), NSD height (Rho=0.100) and width (Rho=0.019), and PED number (Rho=0.016), height (Rho=0.246) and width (Rho=0.066). The serum VEGF-A showed a weak positive correlation (p>0.05) with most OCTA features, whereas it correlated negatively with FAZ area (Rho=-0.136) and perimeter (Rho=-0.128). A month after intravitreal injection, LogMAR BCVA improved to 0.42±0.42, CMT measured 256.65±109.57 microns, and SFCT was 209.84±41.53 microns. The median serum VEGF-A level was 336.31 (IQR 259.455-500.105) pg/ml. Serum VEGF-A showed weak positive correlation with LogMAR value of BCVA (Rho=0.046), CMT (Rho=0.063), SFCT (Rho=0.094), NSD number (Rho=0.290), height (Rho=0.269) and width (Rho=0.289), and PED number (Rho=0.306), height (Rho=0.204) and width (Rho=0.124). The serum VEGF-A showed negative weak correlations (p>0.05) with OCTA features, including FAZ metrics --area (Rho=-0.156), perimeter (Rho=-0.114), and circularity (Rho=-0.335), as well as VD in various retino-choroidal layers: superficial capillary plexus (Rho=-0.282); deep capillary plexus (Rho=-0.225), outer retinal chorio-capillaries (Rho=-0.295), chorio-capillaries (Rho=-0.312) and choroid (Rho=-0.274). Higher serum VEGF-A was associated with worse VA and increased CMT, SFCT, NSD height and width, and PED number, height, and width. An increase in VEGF-A was related to reduced FAZ area and perimeter. At baseline, higher levels of VEGF-A were accompanied by an increase in VD in all layers except the outer retina and choroid. After treatment with intravitreal RbB, a similar pattern was observed; however, increased VEGF-A was found to be coupled with decreased VD in all retino-choroidal layers, except the outer retina.
2026-05-27 | Effects of Selective Retina Therapy on Central Serous Chorioretinopathy with Serous Pigment Epithelial Detachments.
Background/Objective: This study's aim is to evaluate the anatomical and functional effects of selective retina therapy (SRT) in patients with central serous chorioretinopathy (CSC) accompanied by serous pigment epithelial detachment (PED). Methods: This retrospective study included 32 eyes from 32 patients with CSC and serous PED treated with SRT. Pulse energy and micropulse number were adjusted based on test spot visibility on fundus photographs. Best-corrected visual acuity (BCVA; logMAR), central foveal thickness (CFT), subretinal fluid (SRF) height, PED height, and subfoveal choroidal thickness were assessed at baseline and at 1, 2, and 3 months post-treatment. Retinal sensitivity was evaluated using microperimetry at baseline and 3 months. Results: At 3 months after SRT, complete SRF resolution was achieved in 78.1% of eyes (25/32). Mean BCVA improved significantly from 0.29 ± 0.30 logMAR at baseline to 0.20 ± 0.29 logMAR (p = 0.006). Mean CFT decreased from 284.7 ± 91.3 µm to 165.7 ± 94.8 µm (p < 0.001). Mean SRF height decreased from 150.5 ± 74.6 µm to 20.9 ± 48.3 µm (p < 0.001), and mean PED height decreased from 101.7 ± 96.9 µm to 33.3 ± 37.6 µm (p < 0.001). Retinal sensitivity showed a non-significant improvement at 3 months (p = 0.108). Reduction in PED height was moderately correlated with reduction in SRF height (r = 0.446, p = 0.011). Conclusions: SRT was associated with reductions in PED and SRF in CSC. These findings should be interpreted cautiously given the absence of a control group and the potential for spontaneous changes in SRF and PED.
2026-05-27 | Long-term analysis of intravitreal brolucizumab in recalcitrant cases of chronic central serous retinopathy complicated by neovascularization (pachychoroid neovasculopathy).
To assess the long-term safety and efficacy of intravitreal brolucizumab (Pagenax®) in eyes with chronic central serous chorioretinopathy (CSC) complicated by neovascularization (pachychoroid neovasculopathy) refractory to prior therapies. In this single-center retrospective case series (January 2021-July 2025), 30 eyes of 25 patients with recalcitrant and refractory cases of chronic CSC with secondary neovascularization were treated off-label with pro-re-nata intravitreal brolucizumab. Best-corrected visual acuity (BCVA), central subfield thickness (CST), and pigment epithelial detachment (PED) height were measured at baseline and 6 months, with extended outcomes in nine eyes assessed at 1 and 2 years. Adverse events were recorded at each visit. At 6 months, the mean CST decreased significantly from 328 ± 222 µm to 172 ± 79 µm ( P < 0.001), and the PED height declined from 262 ± 192 µm to 155 ± 123 µm ( P = 0.027). BCVA improved or remained stable in 90% of eyes, with no significant change in mean logMAR (0.38 to 0.33, P = 0.314). In the extended cohort, CST reductions persisted at 1 year (412 ± 241 µm to 271 ± 239 µm, P = 0.009) and 2 years (412 ± 241 µm to 164 ± 42 µm, P = 0.004), while BCVA stayed stable. A weak correlation suggested more injections might be linked to slightly poorer BCVA, possibly due to resistant cases requiring more treatment. No adverse events were noted. Intravitreal brolucizumab administered PRN effectively resolves persistent fluid and reduces PED in chronic CSC with PNV refractory to prior treatments, while maintaining stable visual acuity and demonstrating a favorable safety profile through 2 years of follow-up.
gene therapies
2022-08-10 | An Insertion Variant in CRH Confers an Increased Risk of Central Serous Chorioretinopathy.
To identify a novel corticotropin-releasing hormone (CRH) gene variant relevant in patients with central serous chorioretinopathy (CSC). We performed a genetic study of CSC in families and sporadic cases with controls. Using whole-exome sequencing and linkage analysis, we identified a heterozygous insertion variant, Gln52insPro, in the CRH gene that cosegregated in two Chinese families with CSC. This variant was evaluated among an additional 1307 patients with CSC and 1438 ethnicity-matched control individuals from three independent Chinese cohorts. The CRH variant was strongly associated with CSC in these cohorts of Chinese patients (Pmeta = 1.24 × 10-11; odds ratio, 3.01; 95% confidence interval, 2.15-4.21). The risk variant Gln52insPro decreased CRH gene expression. Our results implicate the hypothalamic-pituitary-adrenal stress response system in the pathogenesis of CSC and provide a novel rationale for therapeutic intervention.
2015-11-01 | Selective Retina Therapy
The safety and effectiveness of Selective Retina Therapy as a procedure that induces the regeneration of the retinal pigmented epithelial cells by selectively destroying only the retinal pigmented epithelial cell in patients with retinal diseases were assessed. Assessment was made on the Selective Retina Therapy by extending the range of patients to be subjected to this technology by including macular edema and central serous chorioretinopathy patients. Intervention procedures included a broad range of technologies that selectively damages the retinal pigments and all devices without limitation were selected in assessment of the safety and effectiveness of this technology. For information search, 8 domestic databases including Korea Med and overseas databases including Ovid-Medline, Ovid-Embase and Cochrane Library were used. A total of 55 relevant literatures were searched through the use of search words related to ‘selective retina therapy’. As the result, a total of 7 literatures were included in the final assessment by applying the criteria for selection and exclusion to the 28 literatures after having excluded 27 overlappingly searched literatures. Each of the stages from literature search to application of selection standards and extraction of data were carried out independently by 2 assessors under the deliberation by the Sub-committee. Although there is no safety problem with the Selective Retina Therapy when implemented on central serous chorioretinopathy patient, there is no literature that made report on the key effectiveness variable of the Therapy,making the assessment of the effectiveness of this technology difficulty. Therefore, this technology was assessed to be at a stage that requires further researches (Recommendation rating of D, Classification as technology in research stage i).
2012-01-17 | Suprachoroidal Electrotransfer: A Nonviral Gene Delivery Method to Transfect the Choroid and the Retina Without Detaching the Retina
Photoreceptors and retinal pigment epithelial cells (RPE) targeting remains challenging in ocular gene therapy. Viral gene transfer, the only method having reached clinical evaluation, still raises safety concerns when administered via subretinal injections. We have developed a novel transfection method in the adult rat, called suprachoroidal electrotransfer (ET), combining the administration of nonviral plasmid DNA into the suprachoroidal space with the application of an electrical field. Optimization of injection, electrical parameters and external electrodes geometry using a reporter plasmid, resulted in a large area of transfected tissues. Not only choroidal cells but also RPE, and potentially photoreceptors, were efficiently transduced for at least a month when using a cytomegalovirus (CMV) promoter. No ocular complications were recorded by angiographic, electroretinographic, and histological analyses, demonstrating that under selected conditions the procedure is devoid of side effects on the retina or the vasculature integrity. Moreover, a significant inhibition of laser induced-choroidal neovascularization (CNV) was achieved 15 days after transfection of a soluble vascular endothelial growth factor receptor-1 (sFlt-1)-encoding plasmid. This is the first nonviral gene transfer technique that is efficient for RPE targeting without inducing retinal detachment. This novel minimally invasive nonviral gene therapy method may open new prospects for human retinal therapies.
cell therapies
2026-08-11 | Role of Circulating Immune Cells in Mediating the Effect of Gut Microbiota on Central Serous Chorioretinopathy: A Mendelian Randomization and Mediation Analysis.
Recent observational studies have revealed gut microbiota influences the development and progression of several retinal diseases. However, the causal relationship between gut microbiota and central serous chorioretinopathy (CSCR) are still not understood. The data of gut microbiota and circulating immune cell traits were identifed from large‑scale genome‑wide association studies (GWAS) summary data, and the data of CSCR come from FinnGen database.Mendelian randomization (MR) was used to investigate the causal relationships between gut microbiota, circulating immune cells, and CSCR. Mendelian randomization methods included inverse variance weighting and Bayesian weighting. Cochran's Q, Egger intercept, and MR-PRESSO were used to evaluate heterogeneity, genetic pleiotropy, and horizontal pleiotropy. Sensitivity was evaluated using leave-one-out method.Finally, we explored whether immune cell act as a mediating factor in the pathway from gut microbiota to CSCR. 12 gut microbiota traits were associated with increased genetic risk of CSCR, 2 were associated with decreased genetic risk of CSCR.Mediated mendelian randomization analysis identified 10 pathways through which gut microbiota affects the genetic prediction of CSCR through circulating immune cell mediation, with Demequinaceae via IgD-CD24-% lymphocyte (7.0%) and CD24 on sw mem (3.5%), Demequina via IgD-CD24-% lymphocyte (9.6%), GCA-900066575 sp900066385 via CD4 on CD28 + CD4+ (9.7%), Saccharofermentanaceae via CD45 on HLA DR+T cells (4.7%), CD80 on plasmacytoid DC (5.0%), and CD80 on CD62L + plasmacytoid DC (5.9%), UBA737 via HLA DR+CD8br AC (4.3%), CAG-1000 sp000434555 via Resting Treg% CD4 Treg (3.3%) affecting the genetic risk of CSCR. Our mediated mendelian randomization analysis provides genetic evidence suggesting that circulating immune cells may mediate the causal relationship between gut microbiota and CSCR. The identified associations and mediation effects offer new insights into potential therapeutic avenues for CSCR.
2022-04-21 | Exploring the choroidal vascular labyrinth and its molecular and structural roles in health and disease.
The choroid is a key player in maintaining ocular homeostasis and plays a role in a variety of chorioretinal diseases, many of which are poorly understood. Recent advances in the field of single-cell RNA sequencing have yielded valuable insights into the properties of choroidal endothelial cells (CECs). Here, we review the role of the choroid in various physiological and pathophysiological mechanisms, focusing on the role of CECs. We also discuss new insights regarding the phenotypic properties of CECs, CEC subpopulations, and the value of measuring transcriptomics in primary CEC cultures derived from post-mortem eyes. In addition, we discuss key phenotypic, structural, and functional differences that distinguish CECs from other endothelial cells such as retinal vascular endothelial cells. Understanding the specific clinical and molecular properties of the choroid will shed new light on the pathogenesis of the broad clinical range of chorioretinal diseases such as age-related macular degeneration, central serous chorioretinopathy and other diseases within the pachychoroid spectrum, uveitis, and diabetic choroidopathy. Although our knowledge is still relatively limited with respect to the clinical features and molecular pathways that underlie these chorioretinal diseases, we summarise new approaches and discuss future directions for gaining new insights into these sight-threatening diseases and highlight new therapeutic strategies such as pluripotent stem cell‒based technologies and gene therapy.
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Drug Discovery Landscape
2 orphan drug designations for Central serous chorioretinopathy.
2 orphan drug designations for Central serous chorioretinopathy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Spironolactone | small molecules | EMA | 2026-01-09 | — | Dan Mejlachowicz |
verteporfin | small molecules | FDA | 2012-03-09 | — | Valeant Pharmaceuticals North America LLC |
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