Divisions
College of Physicians and Surgeons (CUMC
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Gene editing system for the treatment of autosomal dominant disease-related genes
CU18283
Mutation-independent CRISPR pair replaces dominant alleles with a functional gene to treat autosomal dominant diseases.
Tags
CRISPR
Gene therapy
Genetic disorder
Inventors
Stephen H. Tsang M.D., Ph.D.
Wen-Hsuan Wu
Yi-Ting Tsai
Manager
Kristin Neuman
Departments
Nutritional and Metabolic Biology
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Faculty of the Arts & Sciences
Release Date
2023-12-28
Collections
Ophthalmology
Gene Therapy
Therapeutic prime editing for gene therapy against ocular dystrophy
CU22243
Prime-editing therapy targets PRPH2 splice mutations (c.828+) for retinal dystrophies, enabling precise in-eye gene corrections.
Tags
Animal husbandry
Cas9
Genome
Inventors
Bruna Lopes da Costa
Peter M.J. Quinn
Salvatore M. Caruso
Manager
Kristin Neuman
Departments
Biomedical Engineering
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2024-04-25
Collections
Ophthalmology
Gene Therapy
Metabolic modulation of cells for neurodegenerative disorder treatment
CU17362
Metabolic reprogramming to boost cellular anabolism, slowing neurodegeneration by targeted glycolytic pathway manipulation.
Tags
Anabolism
Assay
Catabolism
Inventors
Christine Xu
Karen Sophia Park
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2023-04-15
Collections
Ophthalmology
Small Molecule
Gene Therapy
Adeno-associated virus Glut1 gene therapy for rescuing glucose transport in Glut1 deficiency syndrome
CU15054
AAV-based Glut1 gene therapy restores brain glucose transport in Glut1 deficiency, offering a one-time, long-term treatment.
Tags
Adeno-associated virus
Blood–brain barrier
Cognitive deficit
Inventors
Darryl C. De Vivo
Guangping Gao
Kristin Engelstad
Manager
Kristin Neuman
Departments
Neurology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Office of Technology Management
Release Date
2025-08-19
Diagnosis of neuropsychiatric disorders using magnetic resonance imaging
2855
MRI-based pattern-recognition enables highly accurate, early diagnosis of neuropsychiatric disorders by whole-brain morphology.
Tags
Amygdala
Bipolar disorder
Comorbidity
Inventors
Bradley Peterson
Ravi Bansal
Manager
Kristin Neuman
Departments
Psychiatry
Psychiatry, Anatomy and Cell Biology
Divisions
College of Physicians and Surgeons (CUMC
Release Date
2020-03-11
Targeted gene therapy for retinopathies
CU21122
CRB1-targeted gene therapy delivers isoform-specific CRB1 to Müller glia and photoreceptors, treating CRB1-retinopathies.
Tags
Gene expression
Gene therapy
Genetic disorder
Inventors
Peter M.J. Quinn
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2023-07-06
Collections
Ophthalmology
Gene Therapy
Gene therapy for Bestrophin-1 (BEST1) mutations in retinal degenerative diseases
CU21279
AAV BEST1 gene therapy targets dominant BEST1 retinal mutations to restore function and potentially cure associated degeneration.
Tags
Adeno-associated virus
Gene therapy
Genetic disorder
Inventors
Stephen H. Tsang M.D., Ph.D.
Tingting Yang
Yu Zhang
Manager
Kristin Neuman
Departments
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2023-09-08
Collections
Ophthalmology
Gene Therapy
Chop-Stick gene therapy for retinitis pigmentosa and other autosomal dominant ocular diseases
CU15206
Chop-Stick CRISPR therapy edits dominant ocular genes to disable mutations and replace with wild-type function for RP and related diseases.
Tags
Allele
CRISPR
Complementary DNA
Inventors
Lawrence Chan
Stephen H. Tsang M.D., Ph.D.
Wen-Hsuan Wu
Manager
Kristin Neuman
Departments
Nutritional and Metabolic Biology
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Faculty of the Arts & Sciences
Release Date
2022-09-23
Collections
Ophthalmology
Gene Therapy
Gene editing for treatment of retinitis pigmentosa
CU23293
CRISPR-based retinal metabolism reprogramming targets PHD2 to slow retinitis pigmentosa progression, offering broad RP treatment potential.
Tags
Adeno-associated virus
CRISPR
CRISPR gene editing
Inventors
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Release Date
2024-11-26
Collections
Ophthalmology
Alzheimer's disease detection and treatment based on ceramide and sphingolipid levels
CU15147
Gene-targeted reduction of ER-MAM APP-C99 via ceramide/sphingolipid biomarkers enables early Alzheimer’s detection and therapeutic modulation.
Tags
Amyloid beta
Biomarker
Cell membrane
Inventors
Eric A. Schon Ph.D.
Estela Area-Gomez
Manager
Kristin Neuman
Departments
Neurology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2024-04-25
Collections
CNS/Neurology
Universal gene therapy for retinal degeneration
CU18340
Broad, gene-agnostic retinal therapy using AAV-delivered PGC1α/NRF2 or inhibitors to rebalance metabolism, reduce oxidative stress, slow degeneration.
Tags
Antioxidant
Gene therapy
Genetic disorder
Inventors
Stephen H. Tsang M.D., Ph.D.
Xuan Cui
Manager
Kristin Neuman
Departments
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2023-05-15
Collections
Ophthalmology
Gene Therapy
CNS/Neurology
Modified prime editor for gene therapy optimization
CU22084
AAV-compatible, compact prime editor enables single-delivery gene therapy and research tools by packaging smaller genome-editing payloads.
Tags
Achromatopsia
Acid
Adeno-associated virus
Inventors
Bruna Lopes da Costa
Peter M.J. Quinn
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Biomedical Engineering
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2022-09-23
Collections
Gene Therapy
Lecithin cholesterol acyltransferase (LCAT) lipid activators for prevention and treatment of neurodegeneration
CU20145
LCAT-activating lipids aim to restore brain lipid metabolism, potentially slowing neurodegeneration and offering broad neurological disease applications.
Tags
Acyltransferase
Amyloid
Apolipoprotein E
Inventors
Laura Beth Johnson McIntire
Manager
Jerry Kokoshka
Departments
Pathology
Divisions
College of Physicians and Surgeons (CUMC
Release Date
2023-08-04
Collections
Small Molecule
CNS/Neurology
Conditional knockout mouse model to study genomic instability of BRCA1 mutations
CU23027
Inducible BRCA1 exon 2 knockout mouse model to study BRCA1-related genomic instability, tumorigenesis, and therapy screening.
Tags
Ablation
Allele
BRCA1
Inventors
Foon Wu-baer
Richard Baer Ph.D.
Manager
Joan Martinez
Departments
Pathology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2022-10-10
Collections
Oncology
Women's Health
Conditional mouse model for the BARD1 gene
CU23026
A conditional Bard1 mouse model enables tissue-specific gene knockout to study BRCA1/BARD1 cancer risk and therapy.
Tags
Allele
BRCA1
Breast cancer
Inventors
Foon Wu-baer
Richard Baer Ph.D.
Manager
Joan Martinez
Departments
Pathology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2022-10-10
Collections
Oncology
Women's Health
Caspase-2 inhibitors for treating neurodegenerative diseases
CU23390
Non-invasive, selective caspase-2 inhibitors block neurodegeneration, preventing synapse loss and cognitive decline in Alzheimer's models.
Tags
Active site
Amyloid beta
Axon
Inventors
Carol M. Troy MD, PhD
Claire W. Chen
Etienne Jacotot
Manager
Jerry Kokoshka
Departments
Pathology
Pathology & Cell Biology
Taub Institute
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2023-11-17
Collections
CNS/Neurology
Allele-specific prime editing strategies for rhodopsin-mediated retinitis pigmentosa
CU24356
Allele-specific prime editing silences mutant RHO in retinal disease, preserving normal gene function without DNA breaks. 15 words, 139 chars.
Tags
Allele
Gene therapy
Genetic disorder
Inventors
Peter M.J. Quinn
Salvatore M. Caruso
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Biomedical Engineering
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2026-02-14
Regulation of cholesterol efflux for treating macular degeneration
CU21299
Promotes retinal cholesterol efflux via CES1 upregulation or EGFR signaling modulation to slow or prevent macular degeneration.
Tags
Cholesterol
Enzyme
Epidermal growth factor receptor
Inventors
Stephen H. Tsang M.D., Ph.D.
Yi-Ting Tsai
Manager
Kristin Neuman
Departments
Biomedical Engineering
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2021-04-27
Collections
Ophthalmology
Gene Therapy
AVPR1A blockade for the treatment of social isolation-induced anxiety in females
CU21294
AVPR1A-targeted therapy reduces social isolation-induced anxiety in females by modulating AVP signaling in the amygdala.
Tags
Amygdala
Estrogen receptor
Genetically modified mouse
Inventors
Lori M. Zeltser
Marie Louise Pascale Jocelyna Francois
Manager
Kristin Neuman
Departments
Pathology
Pediatrics
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2024-08-22
Conditional Ctip-co mouse strain for studying DNA resection
CU23028
A conditional CtIP knockout mouse model enables full inactivation studies of DNA resection, cancer development, and therapy screening.
Tags
Allele
Cancer
Carcinogenesis
Inventors
Foon Wu-baer
Richard Baer Ph.D.
Manager
Joan Martinez
Departments
Pathology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2022-10-10
Collections
Oncology
Targeting 22q11.2 microdeletions for efficacious schizophrenia treatment
2925
Targeting Mirta22 (Emc10) from 22q11.2 deletions to normalize neuronal wiring and treat schizophrenia cognitive symptoms.
Tags
Cell membrane
Chromosome
Combinatorics
Inventors
Bin Xu
Joseph A. Gogos
Maria Karayiorgou
Manager
Joan Martinez
Departments
Physiology and Cellular Biophysics
Psychiatry
Divisions
College of Physicians and Surgeons (CUMC
Release Date
2014-05-22
Gene editing to treat retinal dystrophies
CU22019
Prime editing corrects CRB1 mutations in retinal dystrophy, offering isoform-independent gene therapy and research tool potential.
Tags
Cell therapy
Gene expression
Gene therapy
Inventors
Bruna Lopes da Costa
Peter M.J. Quinn
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Biomedical Engineering
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2022-10-31
Collections
Ophthalmology
Gene Therapy
Stem cells and stem cell-derived neurons as a model for studying Alzheimer's Disease
2917
Renewable mouse embryonic stem cell–derived neurons model Alzheimer's pathology for high-throughput drug screening and target discovery.
Tags
Amyloid beta
Assay
Drug discovery
Inventors
Laura Beth Johnson McIntire
Natalie Landman
Tae-Wan Kim Ph.D.
Manager
Jerry Kokoshka
Departments
Neurobiology
Pathology
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Release Date
2016-04-22
Partial occlusion device for microvascular anastomosis repair
CU25002
3D-printed partial occlusion device for microvascular repair provides blood-free field, stability, and customizable, fiber-free safety.
Tags
3D printing
Anastomosis
Biocompatibility
Inventors
Yelena Akelina
Yuzhu Huang
Manager
Jerry Kokoshka
Departments
Orthopedic Surgery
School of General Studies
Divisions
College of Physicians and Surgeons (CUMC
School of General Studies
Release Date
2024-09-16
Serotonin targeting in the gut to treat anxiety and depression
CU21281
Gut-targeted serotonin modulation offers safer, pregnancy-safe mood disorder treatment with fewer systemic side effects.
Tags
Attention deficit hyperactivity disorder
Breastfeeding
Central nervous system
Inventors
Kam Leong
Kara Margolis
Mark Ansorge
Manager
Dovina Qu
Departments
Biomedical Engineering
Pediatrics
Psychiatry
Divisions
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2021-04-16
Collections
CNS/Neurology
Women's Health
High-efficiency CRISPR-Cas nuclease
CU25094
Compact, high-efficiency CRISPR-Cas12j nuclease delivers AAV-packagable, 2x faster gene editing with high fidelity for therapeutics.
Tags
CRISPR
Drug discovery
Ex vivo
Inventors
Anders Steen Knudsen
Stephen H. Tsang M.D., Ph.D.
Manager
Kristin Neuman
Departments
Biomedical Engineering
Ophthalmology
Divisions
College of Physicians and Surgeons (CUMC
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2025-12-18
Collections
Ophthalmology
