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 husbandryCas9GenomeInventorsBruna Lopes da CostaPeter 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 Date2024-04-25CollectionsOphthalmologyGene Therapy
Metabolic modulation of cells for neurodegenerative disorder treatmentCU17362Metabolic reprogramming to boost cellular anabolism, slowing neurodegeneration by targeted glycolytic pathway manipulation.TagsAnabolismAssayCatabolismInventorsChristine XuKaren Sophia ParkStephen H. Tsang M.D., Ph.D.ManagerKristin NeumanDepartmentsOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2023-04-15CollectionsOphthalmologySmall MoleculeGene Therapy
Adeno-associated virus Glut1 gene therapy for rescuing glucose transport in Glut1 deficiency syndromeCU15054AAV-based Glut1 gene therapy restores brain glucose transport in Glut1 deficiency, offering a one-time, long-term treatment.TagsAdeno-associated virusBlood–brain barrierCognitive deficitInventorsDarryl C. De VivoGuangping GaoKristin EngelstadManagerKristin NeumanDepartmentsNeurologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Office of Technology ManagementRelease Date2025-08-19
Diagnosis of neuropsychiatric disorders using magnetic resonance imaging2855MRI-based pattern-recognition enables highly accurate, early diagnosis of neuropsychiatric disorders by whole-brain morphology.TagsAmygdalaBipolar disorderComorbidityInventorsBradley PetersonRavi BansalManagerKristin NeumanDepartmentsPsychiatryPsychiatry, Anatomy and Cell BiologyDivisionsCollege of Physicians and Surgeons (CUMCRelease Date2020-03-11
Targeted gene therapy for retinopathiesCU21122CRB1-targeted gene therapy delivers isoform-specific CRB1 to Müller glia and photoreceptors, treating CRB1-retinopathies.TagsGene expressionGene therapyGenetic disorderInventorsPeter M.J. QuinnStephen H. Tsang M.D., Ph.D.ManagerKristin 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 DNAInventorsLawrence ChanStephen H. Tsang M.D., Ph.D.Wen-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
Alzheimer's disease detection and treatment based on ceramide and sphingolipid levelsCU15147Gene-targeted reduction of ER-MAM APP-C99 via ceramide/sphingolipid biomarkers enables early Alzheimer’s detection and therapeutic modulation.TagsAmyloid betaBiomarkerCell membraneInventorsEric A. Schon Ph.D.Estela Area-GomezManagerKristin NeumanDepartmentsNeurologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2024-04-25CollectionsCNS/Neurology
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 virusInventorsBruna Lopes da CostaPeter M.J. QuinnStephen H. Tsang M.D., Ph.D.ManagerKristin 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
Conditional knockout mouse model to study genomic instability of BRCA1 mutationsCU23027Inducible BRCA1 exon 2 knockout mouse model to study BRCA1-related genomic instability, tumorigenesis, and therapy screening.TagsAblationAlleleBRCA1InventorsFoon Wu-baerRichard Baer Ph.D.ManagerJoan MartinezDepartmentsPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2022-10-10CollectionsOncologyWomen's Health
Conditional mouse model for the BARD1 geneCU23026A conditional Bard1 mouse model enables tissue-specific gene knockout to study BRCA1/BARD1 cancer risk and therapy.TagsAlleleBRCA1Breast cancerInventorsFoon Wu-baerRichard Baer Ph.D.ManagerJoan MartinezDepartmentsPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2022-10-10CollectionsOncologyWomen's Health
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 disorderInventorsPeter M.J. QuinnSalvatore M. CarusoStephen H. Tsang M.D., Ph.D.ManagerKristin 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
AVPR1A blockade for the treatment of social isolation-induced anxiety in femalesCU21294AVPR1A-targeted therapy reduces social isolation-induced anxiety in females by modulating AVP signaling in the amygdala.TagsAmygdalaEstrogen receptorGenetically modified mouseInventorsLori M. ZeltserMarie Louise Pascale Jocelyna FrancoisManagerKristin NeumanDepartmentsPathologyPediatricsDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2024-08-22
Conditional Ctip-co mouse strain for studying DNA resectionCU23028A conditional CtIP knockout mouse model enables full inactivation studies of DNA resection, cancer development, and therapy screening.TagsAlleleCancerCarcinogenesisInventorsFoon Wu-baerRichard Baer Ph.D.ManagerJoan MartinezDepartmentsPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2022-10-10CollectionsOncology
Targeting 22q11.2 microdeletions for efficacious schizophrenia treatment2925Targeting Mirta22 (Emc10) from 22q11.2 deletions to normalize neuronal wiring and treat schizophrenia cognitive symptoms.TagsCell membraneChromosomeCombinatoricsInventorsBin XuJoseph A. GogosMaria KarayiorgouManagerJoan MartinezDepartmentsPhysiology and Cellular BiophysicsPsychiatryDivisionsCollege of Physicians and Surgeons (CUMCRelease Date2014-05-22
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 therapyInventorsBruna Lopes da CostaPeter M.J. QuinnStephen H. Tsang M.D., Ph.D.ManagerKristin 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
Stem cells and stem cell-derived neurons as a model for studying Alzheimer's Disease2917Renewable mouse embryonic stem cell–derived neurons model Alzheimer's pathology for high-throughput drug screening and target discovery.TagsAmyloid betaAssayDrug discoveryInventorsLaura Beth Johnson McIntireNatalie LandmanTae-Wan Kim Ph.D.ManagerJerry KokoshkaDepartmentsNeurobiologyPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2016-04-22
Partial occlusion device for microvascular anastomosis repairCU250023D-printed partial occlusion device for microvascular repair provides blood-free field, stability, and customizable, fiber-free safety.Tags3D printingAnastomosisBiocompatibilityInventorsYelena AkelinaYuzhu HuangManagerJerry KokoshkaDepartmentsOrthopedic SurgerySchool of General StudiesDivisionsCollege of Physicians and Surgeons (CUMCSchool of General StudiesRelease Date2024-09-16
Serotonin targeting in the gut to treat anxiety and depressionCU21281Gut-targeted serotonin modulation offers safer, pregnancy-safe mood disorder treatment with fewer systemic side effects.TagsAttention deficit hyperactivity disorderBreastfeedingCentral nervous systemInventorsKam LeongKara MargolisMark AnsorgeManagerDovina QuDepartmentsBiomedical EngineeringPediatricsPsychiatryDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Fu Foundation School of Engineering and Applied Science (SEAS)Release Date2021-04-16CollectionsCNS/NeurologyWomen's Health
High-efficiency CRISPR-Cas nucleaseCU25094Compact, high-efficiency CRISPR-Cas12j nuclease delivers AAV-packagable, 2x faster gene editing with high fidelity for therapeutics.TagsCRISPRDrug discoveryEx vivoInventorsAnders Steen KnudsenStephen H. Tsang M.D., Ph.D.ManagerKristin NeumanDepartmentsBiomedical EngineeringOphthalmologyDivisionsCollege of Physicians and Surgeons (CUMCFu Foundation School of Engineering and Applied Science (SEAS)Release Date2025-12-18CollectionsOphthalmology
