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The Future of Genetic Therapies for Blindness
Expanding to Other Mutations
- RPE65: FDA-approved Luxturna® has restored vision in 90% of trial participants.
- CEP290: CRISPR-based therapies (e.g., EDIT-101) are in Phase I trials.
Technological Advancements
- Dual-Vector Systems: Overcoming AAV’s 4.7 kb payload limit for larger genes like ABCAA.
- Optogenetics: Using light-sensitive proteins to bypass damaged photoreceptors.
Global Initiatives
- IRDiRC (International Rare Diseases Research Consortium): Aiming to approve 200 new therapies by 2027.
- Charity Partnerships: Organizations like Foundation Fighting Blindness fund trials and patient advocacy.
Voices from the Frontlines
Parental Perspectives
Mrs. [Last Name] said, “Gene therapy gave Jace a chance to see his world.” Broadbin emphasized. “We’re now fundraising to treat his other eye.”
Patient Advocacy
LCA support groups highlight the urgency of equitable access. “No child should go blind when treatments exist,” said Mark Dunning, founder of the LCA Family Network.
