Gene editing system for the treatment of autosomal dominant disease-related genesCU18283Mutation-independent CRISPR pair replaces dominant alleles with a functional gene to treat autosomal dominant diseases.TagsCRISPRGene therapyGenetic disorderInventorsStephen H. Tsang M.D., Ph.D.Wen-Hsuan WuYi-Ting TsaiManagerKristin NeumanDepartmentsNutritional and Metabolic BiologyOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Faculty of the Arts & SciencesRelease Date2023-12-28CollectionsOphthalmologyGene Therapy
Therapeutic prime editing for gene therapy against ocular dystrophyCU22243Prime-editing therapy targets PRPH2 splice mutations (c.828+) for retinal dystrophies, enabling precise in-eye gene corrections.TagsAnimal husbandryCas9GenomeInventorsStephen H. Tsang M.D., Ph.D.Bruna Lopes da CostaPeter M.J. QuinnManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Fu Foundation School of Engineering and Applied Science (SEAS)Release Date2024-04-25CollectionsOphthalmologyGene Therapy
Metabolic modulation of cells for neurodegenerative disorder treatmentCU17362Metabolic reprogramming to boost cellular anabolism, slowing neurodegeneration by targeted glycolytic pathway manipulation.TagsAnabolismAssayCatabolismInventorsStephen H. Tsang M.D., Ph.D.Christine XuKaren Sophia ParkManagerKristin NeumanDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2023-04-15CollectionsOphthalmologySmall MoleculeGene Therapy
Targeted gene therapy for retinopathiesCU21122CRB1-targeted gene therapy delivers isoform-specific CRB1 to Müller glia and photoreceptors, treating CRB1-retinopathies.TagsGene expressionGene therapyGenetic disorderInventorsStephen H. Tsang M.D., Ph.D.Peter M.J. QuinnManagerKristin NeumanDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2023-07-06CollectionsOphthalmologyGene Therapy
Gene therapy for Bestrophin-1 (BEST1) mutations in retinal degenerative diseasesCU21279AAV BEST1 gene therapy targets dominant BEST1 retinal mutations to restore function and potentially cure associated degeneration.TagsAdeno-associated virusGene therapyGenetic disorderInventorsStephen H. Tsang M.D., Ph.D.Tingting YangYu ZhangManagerKristin NeumanDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2023-09-08CollectionsOphthalmologyGene Therapy
Chop-Stick gene therapy for retinitis pigmentosa and other autosomal dominant ocular diseasesCU15206Chop-Stick CRISPR therapy edits dominant ocular genes to disable mutations and replace with wild-type function for RP and related diseases.TagsAlleleCRISPRComplementary DNAInventorsStephen H. Tsang M.D., Ph.D.Lawrence ChanWen-Hsuan WuManagerKristin NeumanDepartmentsNutritional and Metabolic BiologyOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Faculty of the Arts & SciencesRelease Date2022-09-23CollectionsOphthalmologyGene Therapy
Gene editing for treatment of retinitis pigmentosaCU23293CRISPR-based retinal metabolism reprogramming targets PHD2 to slow retinitis pigmentosa progression, offering broad RP treatment potential.TagsAdeno-associated virusCRISPRCRISPR gene editingInventorsStephen H. Tsang M.D., Ph.D.ManagerKristin NeumanDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCRelease Date2024-11-26CollectionsOphthalmology
Universal gene therapy for retinal degenerationCU18340Broad, gene-agnostic retinal therapy using AAV-delivered PGC1α/NRF2 or inhibitors to rebalance metabolism, reduce oxidative stress, slow degeneration.TagsAntioxidantGene therapyGenetic disorderInventorsStephen H. Tsang M.D., Ph.D.Xuan CuiManagerKristin NeumanDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2023-05-15CollectionsOphthalmologyGene TherapyCNS/Neurology
Modified prime editor for gene therapy optimizationCU22084AAV-compatible, compact prime editor enables single-delivery gene therapy and research tools by packaging smaller genome-editing payloads.TagsAchromatopsiaAcidAdeno-associated virusInventorsStephen H. Tsang M.D., Ph.D.Bruna Lopes da CostaPeter M.J. QuinnManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Fu Foundation School of Engineering and Applied Science (SEAS)Release Date2022-09-23CollectionsGene Therapy
Allele-specific prime editing strategies for rhodopsin-mediated retinitis pigmentosaCU24356Allele-specific prime editing silences mutant RHO in retinal disease, preserving normal gene function without DNA breaks. 15 words, 139 chars.TagsAlleleGene therapyGenetic disorderInventorsStephen H. Tsang M.D., Ph.D.Peter M.J. QuinnSalvatore M. CarusoManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Fu Foundation School of Engineering and Applied Science (SEAS)Release Date2026-02-14
Regulation of cholesterol efflux for treating macular degenerationCU21299Promotes retinal cholesterol efflux via CES1 upregulation or EGFR signaling modulation to slow or prevent macular degeneration.TagsCholesterolEnzymeEpidermal growth factor receptorInventorsStephen H. Tsang M.D., Ph.D.Yi-Ting TsaiManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCFu Foundation School of Engineering and Applied Science (SEAS)Release Date2021-04-27CollectionsOphthalmologyGene Therapy
Gene editing to treat retinal dystrophiesCU22019Prime editing corrects CRB1 mutations in retinal dystrophy, offering isoform-independent gene therapy and research tool potential.TagsCell therapyGene expressionGene therapyInventorsStephen H. Tsang M.D., Ph.D.Bruna Lopes da CostaPeter M.J. QuinnManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Fu Foundation School of Engineering and Applied Science (SEAS)Release Date2022-10-31CollectionsOphthalmologyGene Therapy
Simultaneous gene silencing and augmentation for restoration of BEST1-associated retinal degenerationCU21170Mutation-agnostic CRISPR/dCas9 silencing plus gene augmentation restores BEST1 channel function in retinal cells.TagsCRISPRCalcium channelChloride channelInventorsStephen H. Tsang M.D., Ph.D.Tingting YangYu ZhangManagerJoan MartinezDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2026-06-05
High-efficiency CRISPR-Cas nucleaseCU25094Compact, high-efficiency CRISPR-Cas12j nuclease delivers AAV-packagable, 2x faster gene editing with high fidelity for therapeutics.TagsCRISPRDrug discoveryEx vivoInventorsStephen H. Tsang M.D., Ph.D.Anders Steen KnudsenManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCFu Foundation School of Engineering and Applied Science (SEAS)Release Date2025-12-18CollectionsOphthalmology
