Gene therapy for inherited retinal disease
Gene therapy and optogenetics for inherited retinal dystrophies.
Papers per year
Each bar is 12 months; the last one ends 17 Oct 2026. New trials in the same years: 2, 7, 14, 9, 6.
All papers from the last 12 months
181 papers (18 Oct 2025 – 17 Oct 2026), newest first. Loading the full list…
- Efficacy, Safety, and Durability of Gene Therapy for Inherited Retinal Diseases in the Pediatric Population: A Scoping ReviewAm J Ophthalmol2026 NovPMID 42537978
- Optogenetic therapy shows early promise for treating retinitis pigmentosaNat Med2026 Oct 15PMID 42881268
- Advancing genetic pathogenicity prediction with three-dimensional proteoform-phenotype analysismedRxiv2026 Oct 9PMID 42885366
- Unilateral subretinal administration of HORA-PDE6β in patients with retinitis pigmentosa harbouring mutations in the PDE6β gene: study protocol of HORA-PDE6β-001 phase I/II clinical trialBMJ Open Ophthalmol2026 Oct 8PMID 42849941
- Advances in precision medicine for the anterior segment: therapeutic applications using CRISPR-Cas9Arch Soc Esp Oftalmol (Engl Ed)2026 Oct 8PMID 42849713Review
- Optogenetic Therapy for Restoring Aspects of Visual FunctionN Engl J Med2026 Oct 8PMID 42842903
- Microperimetry Sensitivity Changes at the Functional Transition Zone and at the EZ Margins in X-Linked Retinitis Pigmentosa Treated With Gene TherapyTransl Vis Sci Technol2026 Oct 5PMID 42847595RCT
- Progress in gene therapy for RPGR-associated X-linked retinitis pigmentosaExpert Opin Biol Ther2026 OctPMID 42733973Review
- Generation of isogenic gene-corrected cell lines from a USH2A-RP patient-derived iPS cell lineStem Cell Res2026 OctPMID 42710431
- Retinal organoids and stem cell therapy for vision restoration: current progress, persistent challenges, and future directionsInt Immunopharmacol2026 Oct 1PMID 42435540Review
More ophthalmology trends
All specialties →Ischaemic optic neuropathy
Non-arteritic anterior ischaemic optic neuropathy, including reports linked to GLP-1 drugs.
Key point: In acute NAION, increased early optic nerve head swelling rates were associated with visual field and visual acuity worsening by month 6.
AI in retinal imaging
Deep learning on fundus photos and OCT, including signs of systemic disease.
Diabetic retinopathy screening
Screening for diabetic retinopathy, including autonomous AI.
Minimally invasive glaucoma surgery
MIGS devices and procedures.
Key point: Phaco-iStent and phaco-KDB both significantly reduced intraocular pressure and glaucoma medication burden with few postoperative complications.
Presbyopia drops
Eye drops that improve near vision in presbyopia.
Dry eye disease
Dry eye disease and its newer treatments.
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How these numbers are counted
PubMed query, searched in titles and abstracts by publication date:
("inherited retinal"[tiab] OR "retinitis pigmentosa"[tiab] OR voretigene[tiab]) AND ("gene therapy"[tiab] OR "gene editing"[tiab] OR optogenetic*[tiab])Trials: ClinicalTrials.gov studies first submitted in each 12-month window that match the same terms. A query can miss synonyms or catch unrelated papers, so treat counts as approximate.