Targeted gene therapy for retinopathies
Search text
About 24 results
(0.10 seconds)
Over 250 genetic mutations that cause retinal degenerative disorders have been identified in the BEST1 gene. The Technology: Gene therapy for retinal degenerative diseases. This technology is a gene therapy for Bestrophin-1 (BEST1) dominant mutations that are associated with certain retinal degenerative diseases.
Unmet Need: Comprehensive therapeutic for patients with retinal degeneration. By targeting metabolic dysregulation and oxidative stress, this technology stalls degeneration in RPE and RG cells in a non-gene-specific manner. This technology is a generally applicable gene therapy approach that restores metabolic dysregulation and treats retinal degenerative diseases regardless of genetic variation.
Unmet Need: Treatment modality available to retinal dystrophy patients with mutations in the CRB1 gene. Although more precise gene editing tools are being developed, there is no therapy commercially available to target specifically CRB1 mutations. This technology is a gene editing methodology for correcting mutations in the CRB1 gene, one of the major cause of inherited retinal dystrophies.
- 10Gene therapy
- 9Mutation
- 9Gene expression
- 8Retina
- 7Macular degeneration
- Add ...
- 24Biology
- 23Biotechnology
- 7Genetic engineering
- 23Branches of biology
- 17Anatomy
- Add ...
- 8Stephen H. Tsang M.D., Ph.D.
- 4Yi-Ting Tsai
- 2Boglarka Racz
- 2Bruna Lopes da Costa
- 2Foon Wu-baer
- Add ...
- 16Kristin Neuman
- 5Joan Martinez
- 2Jerry Kokoshka
- 1Sara Gusik
- Add ...
- 12Ophthalmology
- 5Medicine
- 4Pathology
- 3Biomedical Engineering
- 2Neurology
- Add ...
- 22Columbia University Medical Center (CUMC)
- 10College of Physicians and Surgeons (CUMC
- 3Fu Foundation School of Engineering and Applied Science (SEAS)
- 2Faculty of the Arts & Sciences
- Add ...
- Add ...