Peptide nucleic acid conjugates for altering gene expression in cancerCU16241Selective peptide nucleic acid polymers block cancer gene expression, targeting mutations like BRAFV600E with improved delivery and specificity.TagsGeneBioavailabilityBiosensorInventorsGary Schwartz M.D.Jeffrey Rothman M.D.ManagerJoan MartinezDepartmentsMedicineOncologyDivisionsColumbia University Medical Center (CUMC)Release Date2023-11-03CollectionsGene TherapyOncology
Compact RNA-guided DNA targeting, cleavage, and transcriptional activationCU23100Compact RNA-guided DNA targeting with Cas12/TnpB family enables programmable, compact gene regulation via binding, cleavage, or transcription activation.TagsGeneArtificial intelligenceEscherichia coliInventorsChance MeersSamuel H. SternbergManagerCynthia LangDepartmentsBiochemistry & Molecular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2026-02-17
CRISPR-transposon platform for programmable DNA integrationCU22373Programmable CRISPR-transposon system enables precise, large-dna integrations and endogenous tagging without double-strand breaks.TagsGeneDNA repairEndogenous retrovirusInventorsSamuel H. SternbergManagerCynthia LangDepartmentsBiochemistry & Molecular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2026-01-09CollectionsGenomics & Multi-Omics
Conditional depletion of WDFY3 in murine models of diseaseCU26144Engineered mouse with conditional WDFY3 knockout to study autophagy, mitophagy, efferocytosis, and neurodegenerative disease mechanisms.TagsGeneAmino acidAutism spectrumInventorsAi YamamotoManagerKristin NeumanDepartmentsPhysiology and Cellular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2026-01-08CollectionsCNS/Neurology
Improved specificity of CRISPR-associated transposon (CAST) systemsCU24013Engineered CAST systems boost on-target DNA insertion accuracy, reducing off-target events for RNA-guided, large‑payload genome integration.TagsGeneBiopharmaceuticalCRISPRInventorsJerrin Thomas GeorgeSamuel H. SternbergManagerCynthia LangDepartmentsBiochemistry & Molecular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2026-01-09CollectionsGenomics & Multi-Omics
Chimeric CRISPR-associated transposon system for RNA-mediated DNA integrationCU24119Chimeric CAST enables efficient, RNA-guided DNA insertion without breaks, combining components for larger cargo in cells.TagsGeneCoevolutionDNA repairInventorsGeorge Davis LampeSamuel H. SternbergManagerCynthia LangDepartmentsBiochemistry & Molecular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2026-01-09CollectionsGenomics & Multi-Omics
D1-Cre knock-in mouse for genetic targeting of basal ganglia circuitsCU26035CRISPR knock-in D1-Cre mouse ensures precise, endogenous D1 neuron targeting in basal ganglia for targeted circuit studies.TagsGeneBasal gangliaCre recombinaseInventorsRui CostaManagerKristin NeumanDepartmentsNeuroscienceDivisionsZuckerman Mind Brain Behavior InstRelease Date2025-11-13
A2a-FlpO mouse model for neuroscience studiesCU26036Precise, cell-type specific A2a-FlpO knock-in mice enable orthogonal D1/D2 dopaminergic studies in basal ganglia circuits.TagsGeneBasal gangliaBehavioral neuroscienceInventorsRui CostaManagerKristin NeumanDepartmentsNeuroscienceDivisionsZuckerman Mind Brain Behavior InstRelease Date2025-11-13
Scalable intercellular signaling language for yeastCU18278A scalable GPCR/peptide intercellular language enabling long-range, switchable, high-order yeast signaling networks for multicellular computation.TagsGeneBiotechnologyCell signalingInventorsJames Ronald BrisboisJef BoekeMiguel JimenezManagerJoan MartinezDepartmentsChemistryDivisionsFaculty of the Arts & SciencesRelease Date2026-02-14
Human ALFY knock-in mice for studying neurodegenerative diseaseCU26143Human ALFY knock-in mice enable tissue-specific, controllable hALFY expression to study neurodegenerative protein aggregation.TagsGeneAutophagyCopy number variationInventorsAi YamamotoManagerKristin NeumanDepartmentsPhysiology and Cellular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2026-01-08CollectionsCNS/Neurology
Knock-in mouse lines to target striatal projection neurons and dopaminergic neuronsCU26031Knock-in Cre/FlpO mice precisely target striatal projection and dopaminergic neurons for cell-type specific neuroscience research.TagsGeneAdenosineAdenosine A2A receptorInventorsRui CostaManagerKristin NeumanDepartmentsNeuroscienceDivisionsZuckerman Mind Brain Behavior InstRelease Date2025-11-07
Gut endocrine Rbp2 knockout mouse model for metabolic hormone studiesCU25453Gut-endocrine cell-specific Rbp2 knockout mouse enables retinoid-driven regulation study of gut hormones and metabolic outcomes.TagsGeneAlleleCausal inferenceInventorsRossana M. CalderonManagerJoan MartinezDepartmentsMedicineDivisionsColumbia University Medical Center (CUMC)Release Date2025-10-16
VPS35 floxed mouse model for Alzheimer's drug discoveryCU25395Inducible, tissue-specific VPS35 knockout mouse enabling controlled adult-onset retromer disruption for Alzheimer’s research.TagsGeneBiomarkerCerebrospinal fluidInventorsScott A. Small M.D.ManagerJerry KokoshkaDepartmentsNeurologyDivisionsColumbia University Medical Center (CUMC)Release Date2025-07-31
Conditional 5-HT1A receptor knockout mouse model for studying antidepressant responseCU25138Inducible, cell-type specific 5HT1AR knockout mouse model reveals dentate gyrus roles in antidepressant response and mood regulation.TagsGene5-HT receptor5-HT1A receptorInventorsRene HenManagerKristin NeumanDepartmentsPharmacologyDivisionsColumbia University Medical Center (CUMC)Release Date2025-02-28
Autophagy activating nucleotide sequence for neurodegenerative disease treatmentCU23286Nucleotide sequence boosts ALFY (WDFY3) to enhance autophagic clearance of neurodegenerative protein aggregates.TagsGeneAutophagyDrug discoveryInventorsAi YamamotoKatherine R. CroceManagerKristin NeumanDepartmentsPathology & Cell BiologyPhysiology and Cellular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2024-12-20CollectionsCNS/Neurology
Aldh3a1-Creert mouse lineCU25020Inducible Aldh3a1-CreERT mouse enables tamoxifen-controlled lineage tracing of Aldh3a1-positive pancreatic β-cells for diabetes research.TagsGeneAldehydeAldehyde dehydrogenaseInventorsDomenico Accili M.D.Jinsook Son PhDManagerCynthia LangDepartmentsEndocrinologyMedicineDivisionsColumbia University Medical Center (CUMC)Release Date2024-09-26
Unique human phenotype to define novel gene function for OCRL1CU22138Neuron-specific OCRL1 variant model reveals brain-focused mechanisms in Lowe syndrome; enables targeted therapies, diagnostics, drug screening.TagsGeneCataractCell membraneInventorsBarbara CorneoCecilia SenaCharles LeDucManagerCynthia LangDepartmentsPathologyPediatricsRehabilitation & Regenerative MedicineDivisionsColumbia University Medical Center (CUMC)Release Date2023-03-03CollectionsRare DiseaseCNS/Neurology
High-throughput approach for base editing the kinomeCU24051High-throughput base editing to selectively inhibit kinases endogenously, enabling scalable kinase function studies and drug target discovery.TagsGeneChemical libraryEndogenous retrovirusInventorsNeil Vasan MD, PhDManagerJerry KokoshkaDepartmentsOncologyDivisionsColumbia University Medical Center (CUMC)Release Date2024-07-12
Combinatorial treatment LZTR1 mutant tumors by inhibiting oncogenic substratesCU23141Combinatorial inhibitors target LZTR1-mutant cancers by suppressing EGFR, AXL, and RIT1 to reduce tumor growth.TagsGeneBreast cancerColorectal cancerInventorsAnna LasorellaAntonio IavaroneAram KoManagerKristin NeumanDepartmentsNeurologyPediatricsDivisionsColumbia University Medical Center (CUMC)Release Date2024-06-06
Conditional mouse model for the BARD1 geneCU23026A conditional Bard1 mouse model enables tissue-specific gene knockout to study BRCA1/BARD1 cancer risk and therapy.TagsGeneAlleleBRCA1InventorsFoon Wu-baerRichard Baer Ph.D.ManagerJoan MartinezDepartmentsPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2022-10-10CollectionsOncologyWomen's Health
Conditional knockout mouse model for the Phf6 geneCU22370A tissue-specific Phf6 conditional knockout mouse model for studying Börjeson-Forssman-Lehmann syndrome and hematologic cancers.TagsGeneAcute myeloid leukemiaHigh-throughput screeningInventorsAdolfo A. Ferrando M.D., Ph.D.Teresa Palomero VasquezManagerJoan MartinezDepartmentsInstitute for Cancer GeneticsPathologyDivisionsCollege of Physicians and Surgeons (CUMC)Columbia University Medical Center (CUMC)Release Date2022-08-20
Truncated ATF5, CEBPB, and CEBPD inhibitors for effective cancer treatmentCU21274Short cell-penetrating truncated inhibitors target ATF5/CEBPB/CEBPD, suppressing cancer survival across tumor types.TagsGeneBreast cancerCancerInventorsJames M. AngelastroLloyd Greene PhDQing ZhouManagerJerry KokoshkaDepartmentsMolecular BiosciencesPathologyPathology & Cell BiologyDivisionsColumbia University Medical Center (CUMC)Release Date2023-10-31CollectionsOncology
ATP7B knockout mouse model for Wilson diseaseCU18413ATP7B knockout mouse models Wilson disease, enabling in vivo study of copper accumulation, liver cirrhosis-like pathology, and therapy screening.TagsGeneCopperDrug developmentInventorsDominik HusterOlesia BuiakovaSvetlana LutsenkoManagerSara GusikDepartmentsDepartment of Human GeneticsDivisionsMolecular and Medical GeneticsRelease Date2023-06-28CollectionsRare Disease
TLX1 mouse model recapitulates clinical symptoms of T cell acute lymphoblastic leukemiaCU23147TLX1 transgenic mouse models human T-ALL, mirroring clinical progression to study pathogenesis, microenvironment, and drug responses.TagsGeneAcute lymphoblastic leukemiaAneuploidyInventorsAdolfo A. Ferrando M.D., Ph.D.Teresa Palomero VasquezManagerJoan MartinezDepartmentsInstitute for Cancer GeneticsPathologyDivisionsCollege of Physicians and Surgeons (CUMC)Columbia University Medical Center (CUMC)Release Date2023-04-15CollectionsOncology
Restoration of retromer complex in neurodegenerative diseases via transcriptional regulatorCU18398Target CREB3L2-ATF4 complex to restore retromer function, slowing neurodegenerative progression in AD/PD.TagsGeneDrug developmentEndosomeInventorsClaudio Andre Gouveia Martins RoqueJimena BaleriolaUlrich HengstManagerJerry KokoshkaDepartmentsPathologyTaub InstituteDivisionsColumbia University Medical Center (CUMC)Release Date2023-03-03CollectionsSmall MoleculeCNS/Neurology
Mouse model for studying immune system lineagesCU23013T-bet (Tbx21) floxed mouse model enables tissue-specific T cell knockout to study immune lineages and diseases.TagsGeneAlleleArthritisInventorsNezih CerebSoo Young YangSteven ReinerManagerKristin NeumanDepartmentsMicrobiologyDivisionsColumbia University Medical Center (CUMC)Release Date2022-10-14
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.TagsGeneAblationAlleleInventorsFoon Wu-baerRichard Baer Ph.D.ManagerJoan MartinezDepartmentsPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2022-10-10CollectionsOncologyWomen's Health
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.TagsGeneAlleleCancerInventorsFoon Wu-baerRichard Baer Ph.D.ManagerJoan MartinezDepartmentsPathologyDivisionsCollege of Physicians and Surgeons (CUMCColumbia University Medical Center (CUMC)Release Date2022-10-10CollectionsOncology
Method for disease phenotype prediction from genetic mutationCU21310Phenotype dosage sensitivity: predicts disease phenotypes from LGD mutations via NMD, enabling personalized ASD outcomes and targeted therapeutics.TagsGeneAutism spectrumExonInventorsAndrew ChiangDennis VitkupJiayao WangManagerJoan MartinezDepartmentsBiomedical InformaticsSystems BiologyDivisionsColumbia University Medical Center (CUMC)Release Date2022-09-11CollectionsCNS/NeurologyPlatform/Research Tool
Mouse model to study prelamin A in agingCU22366A knock-in mouse model producing only prelamin A with near-normal lifespan to study long-term effects of farnesylated prelamin A.TagsGeneAdipose tissueEnzymeInventorsHoward WormanYuexia WangManagerJerry KokoshkaDepartmentsMedicineDivisionsColumbia University Medical Center (CUMC)Release Date2022-08-20
Inducible mouse line for cardiac NOX2 inhibitionCU22219Inducible cardiac NRROS-based NOX2 inhibition in mice; tissue-specific ROS control to protect against diet-induced arrhythmias.TagsGeneAntioxidantAtherosclerosisInventorsJohn MorrowManagerJoan MartinezDepartmentsMedicineDivisionsColumbia University Medical Center (CUMC)Release Date2022-02-16CollectionsCardiovascular
BCL6 knockout mouse models of lymphoma developmentCU21195BCL6 knockout mice model lymphoma development and germinal center immunity, enabling in vivo studies of BCL6 function.TagsGeneAlleleAntibodyInventorsRiccardo Dalla-FaveraManagerJoan MartinezDepartmentsPathologyDivisionsColumbia University Medical Center (CUMC)Release Date2021-02-10CollectionsOncology
Small molecule therapeutics to treat treatment-resistant ALL and T-ALLCU19220NT5C2 inhibitors (e.g., HTP-2) boost chemotherapy efficacy in relapsed ALL/T-ALL, enabling combo treatments against resistance.TagsGeneAcute lymphoblastic leukemiaAssayInventorsAdolfo A. Ferrando M.D., Ph.D.Arie ZaskBrent StockwellManagerJoan MartinezDepartmentsBiological SciencesInstitute for Cancer GeneticsProgram in Cellular, Molecular, Structural and Genetic StudiesDivisionsColumbia University Medical Center (CUMC)Faculty of the Arts & SciencesRelease Date2021-02-03CollectionsSmall MoleculeOncology
Disease-in-a-dish model of neuropsychiatric diseases for high-throughput therapeutic screeningCU20125In vitro hiPSC SETD1A-mutant platform enables high-throughput neuropsychiatric drug screening and pathway discovery.TagsGeneAmyotrophic lateral sclerosisAutismInventorsBin XuJoseph A. GogosManagerJoan MartinezDepartmentsPhysiology and Cellular BiophysicsPsychiatryDivisionsColumbia University Medical Center (CUMC)Release Date2020-10-02CollectionsCNS/NeurologyPlatform/Research Tool
Targeting the formation of aggressive cancer-associated fibroblasts for metastasis inhibition in solid tumorsCU21079Computationally maps adipose-derived cells transforming into COL11A1+ aCAFs to block metastasis across solid tumors.TagsGeneAdipocyteAdipose tissueInventorsDimitris AnastassiouKaiyi ZhuLingyi CaiManagerJerry KokoshkaDepartmentsElectrical EngineeringDivisionsFu Foundation School of Engineering and Applied Science (SEAS)Release Date2020-09-23CollectionsOncology
RNA regulation of the SLC2A1 gene for treatment of Glut1 deficiencyCU17177RNA-based therapy using antisense long non-coding RNA to modulate SLC2A1/Glut1 expression for Glut1 deficiency treatment.TagsGeneAnticonvulsantBiomarkerInventorsMaoxue TangUmrao R. Monani Ph.D.ManagerKristin NeumanDepartmentsPathology & Cell BiologyDivisionsColumbia University Medical Center (CUMC)Release Date2019-05-20CollectionsRare DiseaseOphthalmologyGene Therapy
Mouse line with T cell-specific deletion of Blimp-1CU19236Mouse line deletes Blimp-1 specifically in T cells, enabling in vivo study of Blimp-1 function in myeloid lineage.TagsGeneB cellCombinatoricsInventorsKathryn L. Calame Ph.D.Miriam ShelefManagerSara GusikDepartmentsMicrobiologyDivisionsColumbia University Medical Center (CUMC)Release Date2019-04-02
Epigenetic combination therapy to treat germinal center derived lymphomasCU17258Personalized epigenetic combo therapy for EZH2/dysregulated GC-DLBCL; dual EZH2-HDAC inhibition enhances efficacy.TagsGeneChemotherapyCombination therapyInventorsJennifer AmengualJennifer LueOwen O'ConnorManagerJerry KokoshkaDepartmentsMedicineOncologyDivisionsColumbia University Medical Center (CUMC)Release Date2018-01-03CollectionsOncology
Pathogenicity Database for Identification of Disease-Causing Genetic VariationsCU17233TraP scores non-coding and synonymous variants to predict pathogenicity, enabling transcript-focused disease risk assessment and diagnostics.TagsGeneAcidAssayInventorsSahar GelfmanManagerJoan MartinezDepartmentsInstitute for Genomic Medicine (IGM)DivisionsColumbia University Medical Center (CUMC)Release Date2017-09-01
Method to target valproic acid treatment for the negative symptoms of psychiatric disorders using pharmacogenomicsCU15242Personalized valproic acid therapy: predict response via circulating proline and COMT genotype to target treatment and reduce negative symptoms.TagsGeneBipolar disorderCatechol-O-methyltransferaseInventorsCatherine ClellandManagerKristin NeumanDepartmentsPathologyDivisionsColumbia University Medical Center (CUMC)Release Date2016-09-09
Computational software for cancer diagnosis and prognosis2734Computational biomarker tool detects stage-specific gene signatures for cancer prognosis, diagnosis, and metastasis-targeted therapy guidance.TagsGeneAttractorBiomarkerInventorsDimitris AnastassiouManagerJerry KokoshkaDepartmentsElectrical EngineeringDivisionsFu Foundation School of Engineering and Applied Science (SEAS)Release Date2015-06-06
Transgenic mouse line for targeting hepatic stellate cellsCU15191A Cre-lox transgenic mouse labeling hepatic stellate cells via Lrat promoter enables precise gene manipulation and fibrosis research.TagsGeneAblationCre recombinaseInventorsCeline HernandezFrank Anthony CusimanoJorge Matias CavigliaManagerJoan MartinezDepartmentsMedicineDivisionsColumbia University Medical Center (CUMC)Release Date2015-03-06
A PSTPIP1 knockout mouse for use in the study of autoinflammation or PAPA syndromeCU14295PSTPIP1 knockout mice model autoinflammation/PAPA syndrome, enabling drug screening and immune pathway studies in mammals.TagsGeneAcneAdaptive immune systemInventorsStephen P. GoffManagerOrin HerskowitzDepartmentsBiochemistry & Molecular BiophysicsDivisionsColumbia University Medical Center (CUMC)Release Date2014-06-30
Targeting 22q11.2 microdeletions for efficacious schizophrenia treatment2925Targeting Mirta22 (Emc10) from 22q11.2 deletions to normalize neuronal wiring and treat schizophrenia cognitive symptoms.TagsGeneCell membraneChromosomeInventorsBin XuJoseph A. GogosMaria KarayiorgouManagerJoan MartinezDepartmentsPhysiology and Cellular BiophysicsPsychiatryDivisionsCollege of Physicians and Surgeons (CUMCRelease Date2014-05-22
Overcoming drug resistance in relapsed T-ALL patientsCU13051Mutated NT5C2 inhibitors counteract nucleoside-analog resistance in relapsed T-ALL, enabling first-line therapies and broader applicability.TagsGeneAcute lymphoblastic leukemiaAcute myeloid leukemiaInventorsAdolfo A. Ferrando M.D., Ph.D.Raul RabadanTeresa Palomero VasquezManagerJoan MartinezDepartmentsBiomedical InformaticsInstitute for Cancer GeneticsPathologyDivisionsCollege of Physicians and Surgeons (CUMC)Columbia University Medical Center (CUMC)Release Date2013-09-03
Identification of EGFR fusion proteins enables diagnosis and targeted treatment of cancerCU13242EGFR fusion detection enables cancer diagnosis and targeted therapies, improving precision treatment with fusion-specific inhibition.TagsGeneBreast cancerCancerInventorsAnna LasorellaAntonio IavaroneRaul RabadanManagerKristin NeumanDepartmentsBiomedical InformaticsNeurologyPediatricsDivisionsColumbia University Medical Center (CUMC)Release Date2013-06-17
Tetracycline-Controlled System for Targeted Gene Expression in Axin2-Expressing Cells2987Targeted, tetracycline-controlled gene expression in Axin2-expressing cells enables temporal-spatial studies of Wnt signaling in neural development.TagsGeneDevelopment of the nervous systemEmbryonic developmentInventorsFranklin David Costantini Ph.D.ManagerSara GusikDepartmentsGenetics & DevelopmentDivisionsColumbia University Medical Center (CUMC)Release Date2011-08-23
Mouse Model as a Tool for Spatiotemporal and Lineage-Specific Gene Expression2998Transgenic mice enabling tissue-, time-, and lineage-specific gene expression via Cre-loxP and rtTA/tet systems for precise studies.TagsGeneCre recombinaseDoxycyclineInventorsFranklin David Costantini Ph.D.ManagerSara GusikDepartmentsGenetics & DevelopmentDivisionsColumbia University Medical Center (CUMC)Release Date2011-08-26
Mutant Mouse with Modified Axin Gene for Studying Embryogenetic Significance of Protein-Binding Domains2995Mutant mouse lacking Axin’s six C-terminal amino acids reveals indispensable domain for normal embryogenesis; aids Wnt research.TagsGeneAmino acidC-terminusInventorsFranklin David Costantini Ph.D.ManagerSara GusikDepartmentsGenetics & DevelopmentDivisionsColumbia University Medical Center (CUMC)Release Date2011-08-23
VPS26aTg (Hbeta58) Cos Mouse Model for the Investigation of Alzheimer's Disease2997Transgenic mice with Hbeta58 mutation model retromer-related amyloid trafficking to study Alzheimer’s disease mechanisms.TagsGeneAmyloidBrainInventorsFranklin David Costantini Ph.D.ManagerSara GusikDepartmentsGenetics & DevelopmentDivisionsColumbia University Medical Center (CUMC)Release Date2011-10-31
