Evidence & Resources

Vision changes correlate with seasonal light exposure

Personal observation over 2 years

Patient experiences with low-vision aids

Reports from RP community on effectiveness of assistive tools.

Patient community discussion: supplement experiences

Collected experiences from Foundation Fighting Blindness forum on Vitamin A, lutein, and omega-3 supplementation.

jCyte stem cell therapy trial for RP

Phase 2 trial of human retinal progenitor cell therapy for RP patients.

Editas Medicine CRISPR-based gene editing for retinal disease

Early-stage trial exploring CRISPR delivery to retinal cells.

ProQR QR-421a Phase 2/3 trial for USH2A exon 13

Active clinical trial testing AON-based exon skipping for USH2A patients with exon 13 mutations.

Neuroprotective strategies for retinitis pigmentosa: a systematic review

Evaluation of pharmacological neuroprotection approaches across RP subtypes.

Retinal degeneration pathways in Usher syndrome type 2

Molecular pathways leading to photoreceptor death in USH2A-related conditions.

Antisense oligonucleotide therapy for inherited retinal diseases

Overview of AON approaches including exon-skipping strategies for large genes like USH2A.

USH2A mutation mechanisms and structural protein dysfunction

Review of how USH2A mutations affect usherin protein and photoreceptor cell structure.