Gene therapy for ocular disorders
HK40049894BActive Publication Date: 2026-07-10THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
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Patent Information
- Application Number
- HK42021039398
- Authority / Receiving Office
- HK · HK
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2015-12-14
- Filing Date
- 2021-09-24
- Publication Date
- 2026-07-10
AI Technical Summary
Technical Problem
Choroideremia and achromatopsia are retinal diseases characterized by photoreceptor degeneration and visual impairment, with existing treatments lacking effective gene therapy solutions.
Method used
Development of codon-optimized cDNA sequences for Rab Escort Protein-1 (REP-1) and cyclic nucleotide gated channel proteins (CNGA3 and CNGB3) packaged in recombinant adeno-associated virus (rAAV) vectors for targeted gene augmentation therapy.
Benefits of technology
Enhances protein expression and functional recovery in retinal cells, potentially slowing disease progression and improving visual acuity in affected individuals.
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