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948 results

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A spinal motor neuron-specific enhancer for cell-type specific gene expression control in the developing and adult central nervous system

CU22281

Spinal motor neuron–specific enhancer enables AAV-deliverable, cell-type–restricted gene overexpression/knockdown in adults and development.

Tags: Central nervous system
Cholinergic
Gene expression
Inventors: Hynek Wichterle
Tulsi Patel
Manager: Jerry Kokoshka
Departments: Center for Neurobiology & Behavior
Pathology & Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-05-02
Collections: Gene Therapy
CNS/Neurology

Method for disease phenotype prediction from genetic mutation

CU21310

Phenotype dosage sensitivity: predicts disease phenotypes from LGD mutations via NMD, enabling personalized ASD outcomes and targeted therapeutics.

Tags: Autism spectrum
Exon
Gene
Inventors: Andrew Chiang
Dennis Vitkup
Jiayao Wang
Manager: Joan Martinez
Departments: Biomedical Informatics
Systems Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2022-09-11
Collections: CNS/Neurology
Platform/Research Tool

Microneedle array for cross-barrier drug delivery in the eye, ear and central nervous system

CU18171

Biodegradable microneedle arrays enable precise, cross-barrier drug delivery to the eye, ear, and CNS with controlled release.

Tags: 3D printing
Biodegradation
Central nervous system
Inventors: Anil Lalwani
Aykut Aksit
Daniel N. Arteaga
Manager: Dovina Qu
Departments: Mechanical Engineering
Otolaryngology
Divisions: Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2023-01-24

Predicting breast cancer treatment response through PIK3R1 mutation profiling

CU26021

Predicts breast cancer drug response via endogenous kinase inhibition models using PIK3R1 mutations; rapid, high-throughput screening.

Tags: AKT1
Adenine
Breast cancer
Inventors: Neil Vasan MD, PhD
Manager: Jerry Kokoshka
Release Date: 2025-10-09

Targeting NTPDase8 for treating liver ischemia

CU25127

NTPDase8 modulation protects the liver from ischemia-reperfusion injury, offering a potential first-in-class therapy.

Tags: Allotransplantation
Bleeding
Epistasis
Inventors: George Hasko
Taha Kelestemur
Manager: Kristin Neuman
Departments: Anesthesiology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-10-16
Collections: Immunology & Inflammation

Targeting EGFR and IL-6 for the treatment of liver fibrosis and insulin resistance

CU23130

Targeting Treg-derived amphiregulin–EGFR axis and IL-6 to treat liver fibrosis and insulin resistance in MASH.

Tags: Biomarker
Crosstalk
Cytokine
Inventors: Nicholas Arpaia
Thomas Savage
Manager: Cynthia Lang
Departments: Microbiology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-04-24

VRK2 kinase inhibitors for enhanced cancer immunotherapy

CU21245

VRK2 inhibitors boost PD-1 therapy by targeting downstream signaling, enhancing T cell activation and tumor clearance.

Tags: Adenocarcinoma
Bladder cancer
Cancer immunotherapy
Inventors: Adam Mor
Manager: Cynthia Lang
Departments: Rheumatology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-12-17

Lung regeneration method for treatment of lung diseases

CU23015

Selective de-epithelialization with targeted stem cell engraftment regenerates lung tissue, preserving vasculature for disease treatment and modeling.

Tags: Ex vivo
Lung
Mathematical optimization
Inventors: Camilla Predella
Jing Wang
Nicolino Valerio Dorrello
Manager: Joan Martinez
Departments: Biomedical Engineering
Pediatrics
Divisions: Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2023-11-10

Immature myeloid cell activation pathway in inflammation-linked cancer

CU26121

IL-8–driven immature myeloid cell mobilization fuels inflammation-linked GI cancer; targets IMCs for therapy and biomarkers.

Tags: Angiogenesis
Biomarker
Bone marrow
Inventors: Timothy Wang M.D.
Manager: Jerry Kokoshka
Departments: Herbert and Florence Irving Center for Clinical Research
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2026-01-08

Targeting PAG for cancer immunotherapy

CU23123

PAG-targeting antibodies boost T cell activation, inhibit PD-1 signaling, and reduce tumor growth, enabling monotherapy or combo immunotherapy.

Tags: Animal disease model
Cancer immunotherapy
Combination therapy
Inventors: Adam Mor
Manager: Cynthia Lang
Departments: Rheumatology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-04-07

Modulating inflammation by altering 3’ Untranslated Region (3’ UTR) length

CU23300

Modulates inflammation by lengthening 3’ UTRs to control immunogenic dsRNA, enabling tunable neuroinflammation therapies and cancer immunotherapy.

Tags: Cancer immunotherapy
Homeostasis
Immunogenicity
Inventors: Hachung Chung
Heegwon Shin
Tyler Dorrity
Manager: Jerry Kokoshka
Departments: Microbiology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-07-01
Collections: Oncology
CNS/Neurology

Recombinant protein to treat inflammation

CU23248

Recombinant FSTL-1 protein targets and reduces cardiac inflammation, aiding Kawasaki disease research and myocarditis treatment.

Tags: Anti-inflammatory
Atherosclerosis
Biomarker
Inventors: Mark Gorelik
Manager: Sara Gusik
Departments: Pediatrics/Rheumatology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-05-10
Collections: Cardiovascular

Unique human phenotype to define novel gene function for OCRL1

CU22138

Neuron-specific OCRL1 variant model reveals brain-focused mechanisms in Lowe syndrome; enables targeted therapies, diagnostics, drug screening.

Tags: Cataract
Cell membrane
Cilium
Inventors: Barbara Corneo
Cecilia Sena
Charles LeDuc
Manager: Cynthia Lang
Departments: Pathology
Pediatrics
Rehabilitation & Regenerative Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-03-03
Collections: Rare Disease
CNS/Neurology

Exogenous Rac1 analogs to treat acute lung injury

CU23206

Exogenous activated Rac1 (V12Rac1) protects lungs from ALI/ARDS by stabilizing actin and blocking proinflammatory signaling.

Tags: Actin
Acute respiratory distress syndrome
Epithelium
Inventors: Francine Katz
Galina Gusarova
Jahar Bhattacharya
Manager: Jerry Kokoshka
Departments: Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-11-26

Preconditioning cell treatment for immunotherapy

CU17038

Dual hypoxia and inflammatory cytokine preconditioning boosts mesenchymal stem cells’ immunosuppressive potency for more effective immunotherapies.

Tags: Autoimmune disease
Coronary artery disease
Cytokine
Inventors: Gordana Vunjak-Novakovic
Holly Wobma
Mariko Kanai
Manager: Cynthia Lang
Departments: Biomedical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2023-06-03

Transcriptional reprogramming platform for neuroprotection, rejuvenation, and treatment of motor neuron diseases

CU23208

AAV-delivered Islet1/Lhx3 re-expression reprograms motor neurons, restoring youthful resilience to prevent motor neuron degeneration.

Tags: Amyotrophic lateral sclerosis
Gene expression
Gene therapy
Inventors: Emily Rhodes Lowry
Hynek Wichterle
Tulsi Patel
Manager: Jerry Kokoshka
Departments: Center for Neurobiology & Behavior
Pathology & Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-01-18
Collections: Gene Therapy

Rapid assessment of T cell health for enhanced cancer immunotherapy

CU24313

A rapid platform uses biomaterials, imaging, and deep learning to assess T cell functionality and predict proliferation for immunotherapy.

Tags: Algorithm
Assay
Biomaterial
Inventors: Jia Guo
Lance Kam
Nicole Lamanna
Manager: Dovina Qu
Departments: Biomedical Engineering
Medicine
Psychiatry
Divisions: Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2024-12-18

CD58 modulation for improved cancer immunotherapy outcomes

CU21046

CD58 modulation enhances antitumor immunity, overcoming resistance and informing biomarkers for optimized, combinational cancer immunotherapy.

Tags: Antigen presentation
Biomarker
Cancer cell
Inventors: Benjamin Izar MD, PhD
Johannes Melms
Manager: Joan Martinez
Departments: Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-10-17

Non-invasive laser therapy for cytokine modulation and corneal inflammation management

CU18315

Femtosecond laser therapy non-invasively modulates corneal cytokines to reduce inflammation and keratocyte apoptosis, preserving tissue.

Tags: Analgesic
Antibiotic
Apoptosis
Inventors: Sinisa Vukelic
Manager: Dovina Qu
Departments: Mechanical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2024-08-17
Collections: Ophthalmology

Mechanosensitive regulatory T cell induction for improved adoptive immunotherapy

CU23392

Mechanosensitive substrate stiffness boosts stable regulatory T cell induction, improving durability and effectiveness for adoptive immunotherapy.

Tags: Autoimmune disease
Coating
Immune system
Inventors: Lance Kam
Lingting Shi
Manager: Dovina Qu
Departments: Biomedical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2024-12-12

Real-time in situ measurement of electrical impedance of lung tissue

CU22293

Real-time in situ airway impedance device assesses epithelial health, enabling rapid COPD, asthma, and lung disease monitoring.

Tags: Asthma
Biomarker
Cystic fibrosis
Inventors: Gordana Vunjak-Novakovic
Jiawen Chen
Jinho Kim
Manager: Cynthia Lang
Departments: Biomedical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2024-05-29

Lung organoid cells for therapeutic intervention

CU23361

Therapeutic lung regeneration via patient-specific stem cell–derived distal lung organoids with graftable basal and secretory cells.

Tags: Cell therapy
Chronic obstructive pulmonary disease
Coating
Inventors: Hans-Willem Snoeck
Manager: Joan Martinez
Departments: Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-09-08

Targeted delivery of anti-microRNA for the treatment of liver fibrosis

CU21066

Liver-targeted exosomes deliver anti-miR-132 to curb fibrosis, reducing collagen and pro-fibrotic signals with tissue specificity.

Tags: Cell membrane
Collagen
Fibrosis
Inventors: Akiva Mintz
Fatemeh Momen-Heravi
Manager: Joan Martinez
Departments: College of Dental Medicine
Radiology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-05-02

Generation of self-renewed CD4+ T cells for cancer and viral disease immunotherapy

CU24385

Ex vivo culture method yields durable, polyfunctional cytotoxic CD4+ T cells for enhanced cancer and viral immunotherapies.

Tags: Antigen
Cell culture
Cell therapy
Inventors: Mithil Soni
Pawel Muranski
Prabesh Khatiwada
Manager: Jerry Kokoshka
Departments: Columbia Center for Translational Immunology
Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-09-16

Therapy for EGFR-mutant lung cancer

CU22193

Targeting RA signaling to suppress brain relapse in EGFR-mutant lung cancer, boosting TKIs and preventing metastasis.

Tags: Blood–brain barrier
Epidermal growth factor receptor
Estrogen receptor
Inventors: Anup Biswas
Swarnali Acharyya
Manager: Kristin Neuman
Departments: Institute for Cancer Genetics
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-01-04
Collections: Oncology

Host proteases necessary for human parainfluenza virus spread in human lung

CU22326

Identifies HPIV spread proteases; shows serine protease inhibitors (nafamostat) hinder lung infection, offering targeted antiviral strategy.

Tags: Antiviral drug
Enzyme inhibitor
Evolution
Inventors: Anne Moscona
Kyle Stearns
Matteo Porotto
Manager: Kristin Neuman
Departments: Anesthesiology
Pediatrics
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-02-15

Targeting the CD58:CD2 axis for cancer immunotherapy

CU21140

Activates CD28-CD8+ T-cells via CD58:CD2 axis, offering an immunotherapy option for PD-1–unresponsive cancers.

Tags: Antibody
Antigen-presenting cell
CD28
Inventors: Benjamin Izar MD, PhD
Johannes Melms
Patricia Ho
Manager: Joan Martinez
Departments: Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-06-23
Collections: Oncology

Recombinant ILT3 protein-produced antibodies for immunotherapy treatment of cancer

CU15207

Engineered anti-ILT3 antibodies enable targeted cancer immunotherapy, modulating myeloid signals and enhancing immune blockade.

Tags: Acute myeloid leukemia
Allotransplantation
Antibody
Inventors: George Vlad Ph.D.
Nicole Suciu-Foca
Zheng Xu
Manager: Sara Gusik
Departments: Pathology
Divisions: College of Physicians and Surgeons (CUMC)
Columbia University Medical Center (CUMC)
Release Date: 2023-03-08
Collections: Oncology

Small scale screening bioreactor with airway tissues and mucosa for the progression and treatment of lung disease

CU23307

Bioreactor recreating mucus-lined airways ex vivo for real-time, high-throughput modeling and therapeutic screening.

Tags: Biomimetics
Bioreactor
Cell culture
Inventors: Gordana Vunjak-Novakovic
Jinho Kim
Keith Yeager
Manager: Cynthia Lang
Departments: Biomedical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2024-04-08

Pharmacological inhibitors and mouse models of mitochondrial DNA depletion-deletion syndromes (MDDS)

CU23197

Therapeutic mouse models and deoxynucleoside-based treatment restore mtDNA balance in GUK1-related MDDS, enabling mtDNA repair.

Tags: Acute respiratory distress syndrome
Biomarker
Biopharmaceutical
Inventors: Agustin Hidalgo Gutierrez
Jonathan Shintaku
Michio Hirano M.D.
Manager: Kristin Neuman
Departments: Neurology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-11-08
Collections: Rare Disease

Tri-specific human immunodeficiency virus antibodies for immunotherapy

CU22364

Tri-specific HIV antibody targets three epitopes, offering longer-acting, potent immunotherapy to treat and prevent HIV infection.

Tags: Antibody
Antiviral drug
Epitope
Inventors: David Da-i Ho M.D.
Manager: Kristin Neuman
Departments: Physicians & Surgeons
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2022-07-22

Multiplexed T cell profiling for generating engineered regulatory T cell therapies

CU23310

High-throughput platform maps patient TCR–antigen pairs to create CRISPR‑engineered, autologous Tregs for precise local immune suppression.

Tags: Amyotrophic lateral sclerosis
Antigen
CRISPR
Inventors: Elizabeth M. Bradshaw
Neil Shneider
Ryan Hobson
Manager: Joan Martinez
Departments: Molecular Medicine
Neurology
Divisions: College of Physicians and Surgeons (CUMC)
Columbia University Medical Center (CUMC)
Release Date: 2025-01-02

Radiolabeled microtubule ligands for central nervous system (CNS) imaging

CU17295

Brain-penetrant radiolabeled microtubule PET/SPECT tracers enable in vivo CNS imaging for tumors and neurodegenerative monitoring.

Tags: Blood–brain barrier
Brain
Central nervous system
Inventors: Dileep Kumar
J. John Mann
Manager: Jerry Kokoshka
Departments: Psychiatry
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2018-08-13
Collections: CNS/Neurology

Microvasculature-targeted therapeutic AAV vectors for selective brain delivery

CU24146

Brain-endothelium-targeted AAV gene therapy enables non-invasive, selective CNS delivery, treating Glut1DS and neurovascular diseases.

Tags: Adeno-associated virus
Blood–brain barrier
Central nervous system
Inventors: Shashi Kumar
Umrao R. Monani Ph.D.
Manager: Kristin Neuman
Departments: Neurology
Pathology & Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2026-03-17

Tumor-infiltrating lymphocyte and organoid co-culture for patient-specific immunotherapy

CU22128

Ex vivo tumor-lymphocyte co-culture screens patient TILs for tumor killing, guiding pre-treatment immunotherapy choices and cost savings.

Tags: Cell culture
Cytotoxicity
Drug resistance
Inventors: Alana Nguyen
Michael Shen Ph.D.
Manager: Joan Martinez
Departments: Medicine
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2022-08-22
Collections: Oncology
Platform/Research Tool

Targeted pharmacological agents for the prevention of multiple organ failure in hemorrhagic shock patients

CU23263

Localized anti-inflammatory CD39/CD73 therapy to prevent multi-organ failure in hemorrhagic shock by targeted adenosine production.

Tags: Adenosine
Adenosine receptor
Agent-based model
Inventors: George Hasko
Taha Kelestemur
Manager: Kristin Neuman
Departments: Anesthesiology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2024-04-25
Collections: Oncology
Immunology & Inflammation

Convection-enhanced delivery of topotecan and dexamethasone for malignant gliomas

CU25031

Implantable pump enables chronic, targeted brain delivery of topotecan plus dexamethasone via convection-enhanced delivery for glioma.

Tags: Adjuvant therapy
Animal disease model
Blood–brain barrier
Inventors: Jeffrey Bruce
Peter Canoll
Manager: Jerry Kokoshka
Departments: Neurological Surgery
Pathology & Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-08-29

Protein fragment to promote neurogenesis

CU13345

Dominant-negative ATF5 fragment drives differentiation of neuroprogenitors into mature neurons and glial cells, enabling brain regeneration research.

Tags: Astrocyte
Cellular differentiation
Central nervous system
Inventors: James M. Angelastro
Lloyd Greene PhD
Manager: Jerry Kokoshka
Departments: Molecular Biosciences
Pathology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-01-13

PDE5 inhibitor for Alzheimer's treatment

2439

A brain-penetrant PDE5 inhibitor enhances cGMP signaling, reduces amyloid, and supports synaptic plasticity in Alzheimer’s models.

Tags: Amyloid beta
Amyloid plaques
Blood–brain barrier
Inventors: Donald William Landry MD, Ph.D.
Ottavio Arancio MD, PhD
Shi-xian Deng
Manager: Jerry Kokoshka
Departments: Medicine
Pathology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-12-18

Focused ultrasound nanoparticles for targeted imaging and neuroregeneration

CU21224

Ultrasound-activated nanocup theranostics cross BBB for targeted Alzheimer’s imaging and neuroregenerative therapy.

Tags: Amyloid beta
Antibody
Blood–brain barrier
Inventors: Antonios Poulipoulos
Elisa Konofagou
Manager: Dovina Qu
Departments: Biomedical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2025-07-29

Precision modulation of synaptic chaperones for neurodegeneration treatment

CU23056

Precise Hspa8 variant enhances synaptic SNARE function to treat SMA and other neurodegenerative diseases, via gene therapy.

Tags: Amyotrophic lateral sclerosis
Central nervous system
Chaperone (protein)
Inventors: Umrao R. Monani Ph.D.
Manager: Kristin Neuman
Departments: Pathology & Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2023-09-25
Collections: Gene Therapy
CNS/Neurology

Targeting Delta-2 tubulin for the treatment of peripheral neuropathy

CU25119

Delta-2 tubulin as a therapeutic target to prevent or treat chemotherapy-induced peripheral neuropathy, addressing multiple drug classes.

Tags: Analgesic
Anticonvulsant
Antidepressant
Inventors: Francesca Bartolini Ph.D.
Maria Elena Pero
Manager: Kristin Neuman
Departments: Pathology & Cell Biology
Divisions: College of Physicians and Surgeons (CUMC)
Columbia University Medical Center (CUMC)
Release Date: 2025-12-17
Collections: CNS/Neurology

Serum Amyloid A1 antibody to treat leukemias

CU22360

SAA1-targeting antibody blocks bone marrow–driven leukemia progression, aiming to prevent AML/MDS relapse and broaden hematologic cancer treatment.

Tags: Acute lymphoblastic leukemia
Acute myeloid leukemia
Breast cancer
Inventors: Marta Galan-Diez
Stavroula Kousteni
Manager: Jerry Kokoshka
Departments: Herbert and Florence Irving Center for Clinical Research
Physiology and Cellular Biophysics
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-04-10

Probiotic-guided CAR-T cell system for targeted cancer immunotherapy

CU22211

Engineered probiotic-guided CAR-T therapy targets solid tumors, recruiting immune cells and enhancing immunotherapy with tumor-colonizing bacteria.

Tags: Autoimmune disease
Cancer immunotherapy
Chemokine
Inventors: Candice Gurbatri
Jongwon Im
Nicholas Arpaia
Manager: Cynthia Lang
Departments: Biomedical Engineering
Microbiology
Divisions: Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2022-02-04
Collections: Oncology

Conditional knockout mouse model for studying autosomal-dominant optic atrophy

CU26140

A mammalian, cell-type–specific conditional Ssbp1 R107Q mouse model to dissect autosomal-dominant optic atrophy mechanisms.

Tags: Allele
Arginine
Cre recombinase
Inventors: Chyuan-Sheng (Victor) Lin
Nan-Kai Wang
Yunju Tseng
Manager: Kristin Neuman
Departments: Ophthalmology
Pathology & Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2026-01-08

Blood-brain barrier opening and cavitation imaging using a single diagnostic imaging array

CU21007

Single diagnostic ultrasound array opens the blood-brain barrier and images cavitation simultaneously for cost-effective, targeted brain therapy.

Tags: Beamforming
Blood–brain barrier
Cavitation
Inventors: Elisa Konofagou
Mark T. Burgess
Robin Ji
Manager: Dovina Qu
Departments: Biomedical Engineering
Divisions: Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2025-07-29

Functional recovery of human donor lungs for transplantation

CU13138

Localized regional lung regeneration preserves vasculature to revive rejected donor lungs for transplantation.

Tags: Drug discovery
Ex vivo
Extracellular matrix
Inventors: Donald Freytes
Gopal Singh
Gordana Vunjak-Novakovic
Manager: Cynthia Lang
Departments: Biomedical Engineering
Biomedical Informatics
Surgery
Divisions: Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date: 2025-11-20

Combinatorial treatment for amyotrophic lateral sclerosis (ALS)

CU25447

Combinatorial ASO therapy targets 15 dysregulated genes in SOD1-ALS, mitigating neuron death and boosting motor function.

Tags: Amyotrophic lateral sclerosis
Biomarker
Combinatorics
Inventors: George Z. Mentis Ph.D.
JoonHyung Park
Manager: Jerry Kokoshka
Departments: Pathology & Cell Biology
Pathology and Cell Biology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-07-29

Mouse model with impaired bipolar cell function for congenital stationary night blindness

CU26066

A Grm6-missense mouse model mimics human CSNB, enabling targeted gene editing studies and biomarker discovery for therapy.

Tags: Genetic disorder
Genome editing
Laboratory mouse
Inventors: Nan-Kai Wang
Yunju Tseng
Manager: Kristin Neuman
Departments: Ophthalmology
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2025-11-07

Human ALFY knock-in mice for studying neurodegenerative disease

CU26143

Human ALFY knock-in mice enable tissue-specific, controllable hALFY expression to study neurodegenerative protein aggregation.

Tags: Autophagy
Copy number variation
Cre recombinase
Inventors: Ai Yamamoto
Manager: Kristin Neuman
Departments: Physiology and Cellular Biophysics
Divisions: Columbia University Medical Center (CUMC)
Release Date: 2026-01-08
Collections: CNS/Neurology