Slowing or Delaying Vision Loss in Retinitis Pigmentosa (USH2A gene mutation subtype)
Evidence & Resources
10 items total
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.