Topics and Trends in Most Cited Retinal Development and Disorders Papers

Ranked by citations 18 months after publication

Class of 2026 (Papers Published in 2024)

What topics and trends defined most-cited Retinal Development and Disorders research in the Class of 2026?

The Class of 2026 is driven by advanced cellular models, particularly retinal organoids, alongside a major translational push toward therapeutics. Anti-VEGF therapy saw a striking 6-fold increase, reflecting rapid clinical advancements, while inherited dystrophies like retinitis pigmentosa heavily dominated the highest-impact papers.

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Updated September 13, 2026

At a glance

Field
Retinal Development and Disorders
Cohort label
Class of 2026 (2024 publications)
Papers analyzed
3,261
Papers ranked
20
Top topics in ranked papers
Retinal pigment epithelium, Retinal organoid, retinitis pigmentosa
Publication window
Jan 1, 2024 – Dec 31, 2024
Eligibility
Research articles; reviews excluded
Citation window
18 months post-publication
18m citation range
23–118
Data source
OpenAlex · Retrieved September 2026
License
CC BY 4.0

Rankings

20 papers ranked by 18-month citation count

#1 of 3,261
11818m citations

Gene Editing for <i>CEP290</i> -Associated Retinal Degeneration

Eric A Pierce et al.New England Journal of Medicine202410.1056/nejmoa2309915

Eric A PierceMass Eye and Ear, United States

CEP290Inherited retinal degenerationIVS26 variantEDIT-101CRISPR-Cas9 gene editingSubretinal injectionBest-corrected visual acuityFull-field stimulus testingOra–Visual Navigation ChallengeNational Eye Institute Visual Function Questionnaire–25Children's Visual Function Questionnairecone-mediated visionphotoreceptor functionin vivo genome editingPhase 1/2 clinical trial
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#2 of 3,261
5818m citations

Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study

Peter A Campochiaro et al.The Lancet202410.1016/s0140-6736(24)00310-6

Peter A. CampochiaroJohns Hopkins University School of Medicine, United States

RGX-314gene therapyneovascular AMDSubretinal injectionPhase 1/2 clinical trialdose escalationAnti-VEGF therapyAAV8 vectorchoroidal neovascularizationintravitreal injection frequency reductionsustained transgene expressionretinal gene transfer
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#3 of 3,261
4918m citations

Eye-brain connections revealed by multimodal retinal and brain imaging genetics

Bingxin Zhao et al.Nature Communications202410.1038/s41467-024-50309-w

Bingxin Zhao, Hongtu ZhuUniversity of Pennsylvania, United States

retinal imaging biomarkersbrain imaging geneticsmultimodal magnetic resonance imagingprimary visual cortexvisual pathwaysgenetic architecture analysisendophenotypescross-organ genetic analysisgenetic overlapMendelian randomizationAlzheimer's diseaseneurological disordersneuropsychiatric disorderseye-brain connectionsgenetic causal linksintracranial structurebrain structure measuresbrain function measuresgenomic regionsgenetic risk factors
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#4 of 3,261
3918m citations

A highly reproducible and efficient method for retinal organoid differentiation from human pluripotent stem cells

Jade Harkin et al.Proceedings of the National Academy of Sciences202410.1073/pnas.2317285121

Jason S. MeyerIndiana University, United States

Retinal organoidPluripotent stem cellsquick reaggregation methodsBMP signalingforebrain fate specificationCell fate specificationRNA sequencingRetinal developmentorganoid size and shape regulationdefault forebrain fatetranscriptional changeslineage specificationdifferentiation timelinethree-dimensional cellular aggregatesself-organizationtemporal patterningspatial patterning
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#5 of 3,261
3718m citations

Robust reprogramming of glia into neurons by inhibition of Notch signaling and nuclear factor I (NFI) factors in adult mammalian retina

Nguyet Le et al.Science Advances202410.1126/sciadv.adn2091

Seth Blackshaw, Thanh HoangJohns Hopkins University, United States

direct reprogrammingretinal glial cellsMüller gliaNotch signalingRbpjNotch1/2bipolar cellsamacrine cellsneurogenic basic helix-loop-helix (bHLH) factorsnuclear factor I (NFI) factorsNfia/b/xYap overexpressionneurogenic competenceInherited retinal degenerationGlial-to-neuronal conversionMüller glial proliferationNotch effector genes
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#6 of 3,261
3718m citations

6PPD, Not 6PPD-Quinone, Induced Serious Zebrafish Eye Damage by Disrupting the Thyroid Signaling Pathway

Jing Chang et al.Environmental Science & Technology202410.1021/acs.est.4c11264

Bin WanResearch Center for Eco-Environmental Sciences, Chinese Academy of Sciences, China

6PPD6PPD-quinonezebrafish eye damagethyroid signaling pathwaythyroid hormone receptorTR antagonistmicrophthalmusopsin proteincone photoreceptor cell proliferationretinal layer structurephototactic behaviorGH3 celltrβopn1lw1opn1mw1rpe65anr2e3molecular docking
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#7 of 3,261
3618m citations

Comprehensive single-cell atlas of the mouse retina

Jin Li et al.iScience202410.1016/j.isci.2024.109916

Jin LiBaylor College of Medicine, United States

Single-cell RNA sequencingscRNA-seqmouse retinaC57BL/6J micecellular heterogeneityantibody-based magnetic cell sortingrare population enrichmentMouse Retina Cell AtlasMRCAcell type classification138 cell types12 major cell classesdataset integrationCELLxGENEUCSC Cell BrowserBroad Single Cell PortalRetinal cell typesgene expression profiling
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#8 of 3,261
3618m citations

Real-world six-month outcomes in patients switched to faricimab following partial response to anti-VEGF therapy for neovascular age-related macular degeneration and diabetic macular oedema

Grace A Borchert et al.Eye202410.1038/s41433-024-03364-y

Samantha R. De SilvaUniversity of Oxford, United Kingdom

faricimabAnti-VEGF therapyneovascular AMDdiabetic macular edemapartial responsetreatment switchingVisual acuityETDRS letterscentral subfield thicknesstreatment interval extensiontreatment resistanceanatomical responseReal-world outcomesOxford Eye Hospitalretrospective study
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#9 of 3,261
3318m citations

Integrating genetic regulation and single-cell expression with GWAS prioritizes causal genes and cell types for glaucoma

Wenjun Yan, John M Rouhana, Aboozar Monovarfeshani et al.Nature Communications202410.1038/s41467-023-44380-y

Ayellet V. SegrèHarvard University, United States

primary open-angle glaucomaintraocular pressureRGC deathcolocalization analysisMendelian randomizationGWAS lociexpression quantitative trait locisplicing quantitative trait lociGTEx tissuesextracellular matrix organizationSingle-nucleus RNA sequencingaqueous outflow pathwaysoptic nerve headperipapillary sclerachoroidIOP-dependent mechanismsIOP-independent mechanismsvascular developmentcell adhesion pathways
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#10 of 3,261
3218m citations

RPE melanin and its influence on the progression of AMD

Mary Kaufmann et al.Ageing Research Reviews202410.1016/j.arr.2024.102358

Zongchao HanUniversity of North Carolina, United States

RPE melaninAMD progressionRetinal pigment epitheliumage-related macular degenerationmelanin photoprotectionoxidative stresslipofuscin accumulationphotoreceptor degenerationBruch's membranedrusen formationmelanin degradationphagocytosisvisual cyclegeographic atrophychoroidal neovascularization
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#11 of 3,261
2818m citations

Retinal detachment

Jonathan B Lin et al.Nature Reviews Disease Primers202410.1038/s41572-024-00501-5

Rajendra S. ApteMassachusetts Eye and Ear, United States

Retinal detachmentvitrectomy surgeryscleral bucklepneumatic retinopexyposterior vitreous detachmentretinal tearsproliferative vitreoretinopathyVisual acuityreattachment rate
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#12 of 3,261
2818m citations

Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study

Paul Yang et al.The Lancet202410.1016/s0140-6736(24)01447-8

Paul Yang, Artur V. CideciyanOregon Health & Science University, United States

ATSN-101Leber congenital amaurosisRetinal guanylate cyclasebiallelic mutationsPhase 1/2 clinical trialdose escalationunilateral administrationgene therapyInherited retinal degenerationSubretinal injectionvisual functionretinal structureadeno-associated viral vector
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#13 of 3,261
2618m citations

Senolytic and senomorphic agent procyanidin C1 alleviates structural and functional decline in the aged retina

Yidan Liu, Xiuxing Liu, Xuhao Chen, Zhenlan Yang et al.Proceedings of the National Academy of Sciences202410.1073/pnas.2311028121

Yidan Liu, Xiuxing Liu, Xuhao Chen, Zhenlan Yang, Yehong Zhuo, Wenru SuSun Yat-sen University, China

procyanidin C1senolytic agentssenomorphic agentscellular senescencesenescence-associated secretory phenotypeaged retinaretinal structural declineretinal functional declineSingle-cell RNA sequencingscRNA-seqmouse models of natural agingretinal landscapeSenescent cell accumulationage-related retinal disordersretinal impairment
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#14 of 3,261
2618m citations

Disease Activity and Therapeutic Response to Pegcetacoplan for Geographic Atrophy Identified by Deep Learning-Based Analysis of OCT

Ursula Schmidt-Erfurth et al.Ophthalmology202410.1016/j.ophtha.2024.08.017

Ursula Schmidt‐ErfurthMedical University of Vienna, Austria

pegcetacoplangeographic atrophydeep learning segmentationOptical coherence tomographyphotoreceptor degenerationretinal pigment epithelium lossellipsoid zone lossEZ-RPE differenceage-related macular degenerationOAKS trialDERBY trialtherapeutic response stratificationdisease activity biomarkercomplement inhibitionlongitudinal morphological analysisphase 3 trialRPE loss growth rateEZ loss growth ratebaseline EZ-RPE quartiles
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#15 of 3,261
2618m citations

An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species

Shuang Luo, Hao Jiang et al.Nature Communications202410.1038/s41467-024-48221-4

Xiao Ke, Qiang Zheng, Xun SunChengdu Origen Biotechnology Co. Ltd, China

AAVv128adeno-associated virus capsidsuprachoroidal injectionphotoreceptor transductionRetinal pigment epitheliumintraocular injectionAnti-VEGF therapylaser-induced choroidal neovascularizationneovascular AMDnonhuman primatecryo-EM structureAAV binding residuesnuclear uptakeendosome escaperetinal tissue distributionocular gene deliveryGrade IV lesion suppression
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#16 of 3,261
2418m citations

Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study

M. Dominik Fischer et al.Biomolecules202410.3390/biom14010122

M. Dominik FischerUniversity of Tübingen, Germany

voretigene neparvovecRPE65-mediated inherited retinal dystrophygene therapyPERCEIVE studyregistry-based observational studypost-authorization safety studychorioretinal atrophyOcular inflammationFull-field stimulus testingVisual acuitytreatment-emergent adverse eventsReal-world outcomesviable retinal cellsocular TEAEs
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#18 of 3,261
2418m citations

Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa

Michel Michaelides et al.American Journal of Ophthalmology202410.1016/j.ajo.2024.05.034

Michel MichaelidesUCL Institute of Ophthalmology, United Kingdom

AAV5-hRKp.RPGRbotaretigene sparoparvovecRPGRX-linked retinitis pigmentosaXLRPgene therapydose escalationSubretinal injectionstatic perimetryMicroperimetryvision-guided behaviorRetinal sensitivityRetinal detachmentuveitiscorticosteroid treatmentfunctional vision outcomespediatric patientsrandomized immediate versus deferred treatmentPhase 1/2 clinical trial
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#19 of 3,261
2318m citations

Single-cell analyses reveal transient retinal progenitor cells in the ciliary margin of developing human retina

Birthe Dorgau, Joseph Collin et al.Nature Communications202410.1038/s41467-024-47933-x

Rachel Queen, Majlinda LakoNewcastle University, United Kingdom

Retinal progenitor cellsciliary marginhuman retinal developmentSingle-cell RNA sequencingSingle-cell ATAC sequencingspatial transcriptomicsearly retinal progenitorslate retinal progenitorstransient neurogenic progenitorsretinal neuron differentiationlineage trajectory analysismidgestationpost-conception week 8transcriptional enhanced associate domainTEAD transcription factor binding motifsRetinal organoidcycling progenitorsCell fate specification
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#20 of 3,261
2318m citations

Spectrum of Genetic Variants in the Most Common Genes Causing Inherited Retinal Disease in a Large Molecularly Characterized United Kingdom Cohort

Siying Lin et al.Ophthalmology Retina202410.1016/j.oret.2024.01.012

Gavin ArnoUniversity College London, United Kingdom

Inherited retinal degenerationABCA4USH2ARPGRPRPH2BEST1gene-directed therapieswhole genome sequencingwhole exome sequencingGene panel testingcomplex disease allelesUSH2A exon 13 variantsRPGR ORF15 regionCNGB3BBS1TIMP3EFEMP1RP1founder variantsGenomics England PanelApp
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Dominant research themes and year-over-year shifts in Retinal Development and Disorders

What Topics Define the Class of 2026?

The Class of 2026 in Retinal Development and Disorders is defined by a strong focus on cellular models and specific disease states. Retinal pigment epithelium and retinal organoids emerge as the most prominent topics, reflecting the field's ongoing reliance on advanced in vitro models for studying retinal biology and disease mechanisms. Retinitis pigmentosa is the most highly featured disorder, indicating a major clinical focus on inherited retinal dystrophies. Additionally, therapeutic strategies like anti-VEGF therapy and gene therapy play a significant role, highlighting the translational emphasis of recent high-impact publications.

Leading research themes
Leading research themes

How Did Topics Shift from the Class of 2025 to the Class of 2026?

The transition from the Class of 2025 to the Class of 2026 reveals a pronounced shift toward targeted therapeutics. Notably, anti-VEGF therapy experienced the most dramatic rise, with a 6-fold increase in frequency among top papers, reflecting its expanding applications and new treatment regimens. Retinitis pigmentosa and neovascular AMD also saw significant growth, underscoring an increased emphasis on both inherited and age-related retinal diseases. Conversely, foundational topics such as single-cell RNA sequencing and retinal ganglion cells saw a relative decrease in prominence, suggesting a maturation of basic transcriptomic mapping efforts in favor of translational and clinical applications.

How topics shifted year over year
How topics shifted year over year
Methodology

PRI identifies high-impact research using a transparent, topic-agnostic framework applied consistently across scientific domains. Bibliographic records are drawn from OpenAlex, including publication dates, citation relationships, and document types.

This ranking covers the Class of 2026 cohort: journal articles published in 2024. Reviews and other non-article document types are excluded to ensure comparability.

Research impact is quantified with an 18-month post-publication citation window—the number of citing works published within 18 months of each paper's publication date. This metric captures early impact while controlling for publication age.

An LLM-based relevance classifier then reviews each candidate's title and abstract to confirm substantive alignment with the target domain. Only papers classified as relevant appear in the final ranking.

Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459

Full methodologyGitHubbioRxiv preprint

Cite this ranking

Pepkio Research Index (PRI). Topics and Trends in Most Cited Retinal Development and Disorders Papers, Class of 2026. https://pri.pepkio.com/top-papers/retinal-development-and-disorders/2026. Accessed 2026-10-24.

Methodology
Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459