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Biology
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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 metabolic dysfunction-associated steatohepatitis (MASH)
CU25230
Gene therapy restoring retromer activity to treat MASH by normalizing lipid metabolism and reducing fibrosis risk.
Tags
Cirrhosis
Drug discovery
Fibrosis
Inventors
Gregory A. Petsko
Scott A. Small M.D.
Manager
Jerry Kokoshka
Departments
Ann Romney Center for Neurologic Diseases
Neurology
Divisions
Columbia University Medical Center (CUMC)
Harvard Medical School and Brigham & Women’s Hospital
Release Date
2025-07-31
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
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
Combined
small
molecule
treatment as a synergistic epigenetic
therapy
for pancreatic cancer
CU23218
Combined HDAC and eIF4A inhibition reprograms pancreatic cancer epigenetically, boosting efficacy and tumor suppression in vivo.
Tags
Adenocarcinoma
Apoptosis
Chemotherapy
Inventors
Antonio Fojo
Maryam Safari
Susan Bates
Manager
Jerry Kokoshka
Departments
Medicine
Oncology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2024-08-22
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
Conformational DNA aptamers enabling
small
-
molecule
lateral flow assays
CU26117
Conformational DNA aptamers enable rapid, cost-effective small-molecule detection in lateral flow assays via structure-switching binding.
Tags
Analyte
Antibody
Aptamer
Inventors
Kyungae Yang
Manager
Jerry Kokoshka
Departments
Medicine
Divisions
Columbia University Medical Center (CUMC)
Release Date
2025-12-01
Nanofibrous scaffold for controlled release of
small
molecule
immunomodulators
CU24040
Nanofibrous scaffold locally releases ACHP to inhibit NF-kB, enabling targeted immunomodulation with reduced systemic effects.
Tags
Autoimmune disease
Biomaterial
Drug delivery
Inventors
Fereshteh Zandkarimi
Hannah Childs
Helen Lu Ph.D.
Manager
Joan Martinez
Departments
Biological Sciences
Biomedical Engineering
Divisions
Faculty of the Arts & Sciences
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2023-12-18
Collections
Small Molecule
Small
molecule
activators for preventing vision loss in Best vitelliform macular dystrophy
CU25176
Small molecule activators restore Best1 function to slow BVMD vision loss; targeted, potentially non-invasive retinal therapy.
Tags
4-Aminobenzoic acid
Drug development
Gene therapy
Inventors
Bingchen Yu
Tingting Yang
Yu Zhang
Manager
Joan Martinez
Departments
Chemistry
Ophthalmology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2025-05-01
Collections
Ophthalmology
Gene
therapy
for thymidine kinase 2 (TK2) deficiency
CU18199
Engineered AAV9 gene therapy replaces TK2 to treat TK2 deficiency mitochondrial diseases, potentially correcting DNA depletion and symptoms.
Tags
Comorbidity
Enzyme
Gene therapy
Inventors
Carlos Lopez-Gomez
Hasan Orhan Akman
Michio Hirano M.D.
Manager
Kristin Neuman
Departments
Neurology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2023-01-17
Collections
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
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
Topical
gene
therapy
for the treatment of AP-1-associated genetic skin disorders
CU26038
Topical gene therapy restores AP-1 function in skin, enabling localized, repeatable treatment for monogenic ichthyosis and keratoderma.
Tags
AP-1 transcription factor
Carcinogenesis
Combination therapy
Inventors
Stephen Kaler
Tarun Kaniganti
Manager
Kristin Neuman
Departments
Pediatrics
Divisions
Columbia University Medical Center (CUMC)
Release Date
2026-02-26
Small
molecule
inhibitors of the ID2/VHL interaction
CU17182
Four small molecules block ID2/VHL, promote HIF degradation, potentially slowing tumor growth and angiogenesis in cancers.
Tags
Angiogenesis
Cell growth
Chemotherapy
Inventors
Anna Lasorella
Antonio Iavarone
Dan Sherman
Manager
Kristin Neuman
Departments
Institute for Cancer Genetics
Neurology
Pediatrics
Divisions
Columbia University Medical Center (CUMC)
Release Date
2023-05-19
Collections
Small Molecule
Oncology
Small
molecule
inhibitor of cognitive impairment associated with neurodegenerative disease
CU23096
Small molecule minaprine analog targets 5-HT2B to curb cognitive/behavioral deficits in neurodegenerative diseases, including Alzheimer's.
Tags
5-HT receptor
5-HT2B receptor
Amyloid beta
Inventors
Daniel M. Watterson
Erica Acquarone
Ottavio Arancio
Manager
Jerry Kokoshka
Departments
Pathology
Pharmacology
Taub Institute
Divisions
Columbia University Medical Center (CUMC)
Feinberg School of Medicine
Release Date
2025-04-10
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
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
Small
molecule
treatment for MAPK-regulated cancers
CU20013
MAP4K2-targeted small molecule inhibits MAPK signaling to curb multiple myeloma growth, offering therapy for resistant hematologic cancers.
Tags
Apoptosis
B cell
Cell proliferation
Inventors
Donald William Landry MD, Ph.D.
Jing Fu Ph.D.
Shi-xian Deng
Manager
Jerry Kokoshka
Departments
Medicine
Oncology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2022-08-05
Collections
Small Molecule
Oncology
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
Parameters to maximize nebulized
drug
delivery
to larynx
CU24050
Patient-specific CT-based nebulization parameters optimize aerosol size and velocity to maximize laryngeal drug deposition.
Tags
Aerosol
Drug delivery
Hypersensitivity
Inventors
Michael Pitman MD
Manager
Cynthia Lang
Departments
Otolaryngology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2024-07-05
Multi-lumen microneedle for intracochlear
drug
delivery
and sampling
CU21117
Ultra-sharp multi-lumen microneedles enable simultaneous intracochlear drug delivery and perilymph sampling with zero net volume change.
Tags
Cochlea
Diffusion
Drug delivery
Inventors
Anil Lalwani
Aykut Aksit
Jeffrey W. Kysar
Manager
Dovina Qu
Departments
Mechanical Engineering
Otolaryngology
Divisions
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2024-09-26
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
Computational tool to predict RNA binding protein targets for
gene
therapy
and agricultural bioengineering
CU23342
PPRDecoder: a genome-wide, unbiased algorithm predicting RNA targets of PPR proteins, accelerating gene regulation research.
Tags
Algorithm
Biological engineering
Biotechnology
Inventors
Chaolin Zhang PhD
Manager
Joan Martinez
Departments
Biochemistry & Molecular Biophysics
Divisions
Columbia University Medical Center (CUMC)
Release Date
2023-08-04
Collections
Gene Therapy
Machine learning tool for diagnosis of cutaneous
T
-
cell
lymphoma (CTCL)
CU24366
AI-powered CTCL diagnosis tool links genetic markers with EHR data to improve accuracy, staging, and prognosis.
Tags
Assay
Cancer
Cutaneous T cell lymphoma
Inventors
Larisa Geskin
Manager
Cynthia Lang
Departments
Dermatology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2025-01-30
Ultrasound-responsive microcapsules for localized and on-demand
drug
delivery
CU22072
Ultrasound-triggered, biphasic microcapsules enable on-demand, localized drug release with reduced systemic toxicity and long-term payload stability.
Tags
Chemotherapy
Dextran
Drug delivery
Inventors
Margaret Jakus
Rachel D. Field
Samuel Sia Ph.D.
Manager
Dovina Qu
Departments
Biomedical Engineering
Divisions
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2023-07-20
Highly efficient
T
cell
base editing for enhanced immunotherapies
CU24092
Ultra-efficient base editing in human T cells using adenine/cytosine editors; enhances CAR-T, autoimmune therapies with lower toxicity.
Tags
Adenine
Cancer
Chimeric antigen receptor T cell
Inventors
Benjamin Izar MD, PhD
Zachary Walsh
Manager
Joan Martinez
Departments
Medicine
Medicine - Hematology/Oncology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2024-11-08
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
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
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
Calibration-free affinity biosensors for rapid, accurate
biomarker
detection
CU22223
Calibration-free graphene FET biosensor uses aptamer kinetics for rapid, device-wide reproducible biomarker detection without per-sensor calibration.
Tags
Aptamer
Biomarker
Biosensor
Inventors
Milan N. Stojanovic M.D.
Qiao Lin
Wenting Dai
Manager
Dovina Qu
Departments
Mechanical Engineering
Medicine
Divisions
Columbia University Medical Center (CUMC)
Fu Foundation School of Engineering and Applied Science (SEAS)
Release Date
2025-08-21
Small
molecule
therapeutics to treat treatment-resistant ALL and
T
-ALL
CU19220
NT5C2 inhibitors (e.g., HTP-2) boost chemotherapy efficacy in relapsed ALL/T-ALL, enabling combo treatments against resistance.
Tags
Acute lymphoblastic leukemia
Assay
Cancer cell
Inventors
Adolfo A. Ferrando M.D., Ph.D.
Arie Zask
Brent Stockwell
Manager
Joan Martinez
Departments
Biological Sciences
Institute for Cancer Genetics
Program in Cellular, Molecular, Structural and Genetic Studies
Divisions
Columbia University Medical Center (CUMC)
Faculty of the Arts & Sciences
Release Date
2021-02-03
Collections
Small Molecule
Oncology
Biomarker
and treatment for autoimmune hemolytic anemia (AIHA)
CU24097
Biomarker predicts AIHA onset; apyrase therapy prevents/mitigates AIHA in preclinical models by modulating purinergic signaling.
Tags
Anemia
Autoantibody
B cell
Inventors
Flavia Dei Zotti
Krystalyn Hudson
Manager
Jerry Kokoshka
Departments
Pathology & Cell Biology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2023-12-18
Collections
Rare Disease
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
Preclinical models of angioimmunoblastic
T
-
cell
lymphoma for therapeutic investigation
CU23322
RhoA G17V-driven mouse model of angioimmunoblastic T-cell lymphoma enables translational drug screening and pathogenesis study.
Tags
Cancer cell
Chemotherapy
Gene expression
Inventors
Adolfo A. Ferrando M.D., Ph.D.
Teresa Palomero Vasquez
Manager
Joan Martinez
Departments
Institute for Cancer Genetics
Pathology
Divisions
College of Physicians and Surgeons (CUMC)
Columbia University Medical Center (CUMC)
Release Date
2023-06-15
Collections
Oncology
CRISPR deletion of endogenous
T
cell
receptor chains for optimized immunotherapies
CU20245
CRISPR deletes endogenous TCR chains while preserving transgenic TCRs, enabling safer, off-the-shelf CAR-T/TCR therapies.
Tags
Cancer
Chimeric antigen receptor T cell
Graft-versus-host disease
Inventors
Megan Sykes M.D.
Mohsen Khosravi Maharlooei
Remi Creusot
Manager
Jerry Kokoshka
Departments
Medicine
Divisions
Columbia University Medical Center (CUMC)
Release Date
2025-01-30
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
T
cell
co-receptor clustering for cancer treatment
CU22398
Bispecific SLAMF6 antibodies compartmentalize T cells to enhance activation, enabling targeted cancer immunotherapy with fewer off-target effects.
Tags
Cancer immunotherapy
Co-receptor
Immunotherapy
Inventors
Adam Mor
Manager
Cynthia Lang
Departments
Rheumatology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2023-09-05
Collections
Oncology
High-throughput method for
gene
insertion
CU19411
A high-throughput, vector-based CRISPR system enabling parallel, precise DNA insertions in human cells for scalable genome editing.
Tags
Bacteria
Cas9
Gene therapy
Inventors
Alejandro Chavez
Brijesh Kumar Singh
Manager
Cynthia Lang
Departments
Pathology & Cell Biology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2025-07-17
TLX1 mouse model recapitulates clinical symptoms of
T
cell
acute lymphoblastic leukemia
CU23147
TLX1 transgenic mouse models human T-ALL, mirroring clinical progression to study pathogenesis, microenvironment, and drug responses.
Tags
Acute lymphoblastic leukemia
Aneuploidy
Aortic dissection
Inventors
Adolfo A. Ferrando M.D., Ph.D.
Teresa Palomero Vasquez
Manager
Joan Martinez
Departments
Institute for Cancer Genetics
Pathology
Divisions
College of Physicians and Surgeons (CUMC)
Columbia University Medical Center (CUMC)
Release Date
2023-04-15
Collections
Oncology
Method of
biomarker
testing for autism spectrum disorder for early intervention
CU23349
Biomarker-based newborn metabolomic profiling enables early ASD risk identification and intervention, enhancing timely, personalized treatment.
Tags
Applied behavior analysis
Autism spectrum
Blood plasma
Inventors
Camilla Stoltenberg
Oliver Fiehn
W. Ian Lipkin M.D.
Manager
Kristin Neuman
Departments
Biostatistics
Epidemiology
Molecular and Cellular Biology
Divisions
Mailman School Of Public Health (MSPH)
Technology Transfer Services
Release Date
2024-02-15
Collections
CNS/Neurology
Medical Device
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
Universal
gene
editing strategy for cardiac arrhythmia
CU24243
Universal gene-editing of Rad phosphorylation sites to curb adrenergic CaV1.2 signaling, offering targeted, long-term arrhythmia therapy.
Tags
Arrhythmia
Beta blocker
Fight-or-flight response
Inventors
Gregory Newby
Guoxia Liu
Steven Marx
Manager
Joan Martinez
Departments
Medicine
Divisions
Columbia University Medical Center (CUMC)
Release Date
2026-02-14
Peptide nucleic acid conjugates for altering
gene
expression in cancer
CU16241
Selective peptide nucleic acid polymers block cancer gene expression, targeting mutations like BRAFV600E with improved delivery and specificity.
Tags
Bioavailability
Biosensor
Cell culture
Inventors
Gary Schwartz M.D.
Jeffrey Rothman M.D.
Manager
Joan Martinez
Departments
Medicine
Oncology
Divisions
Columbia University Medical Center (CUMC)
Release Date
2023-11-03
Collections
Gene Therapy
Oncology
Mouse line as a model for Peripheral
T
-
cell
Lymphoma with GATA3+ features
CU23321
Mouse model for Vav1-Myof1-driven Peripheral T-cell Lymphoma (GATA3+) enabling targeted therapy screening and pathogenesis study.
Tags
CD4
Carcinogenesis
Genetic testing
Inventors
Adolfo A. Ferrando M.D., Ph.D.
Teresa Palomero Vasquez
Manager
Joan Martinez
Departments
Institute for Cancer Genetics
Pathology
Divisions
College of Physicians and Surgeons (CUMC)
Columbia University Medical Center (CUMC)
Release Date
2023-06-15
Collections
Oncology
ARP-154 (HeLa T4+) human cancer
cell
line generated by
gene
transfer for AIDS research
CU24290
CD4-expressing HeLa-derived cell line enables AIDS research; facilitates HIV binding studies, drug screening, and therapeutic development.
Tags
AIDS
Antigen
Autoimmune disease
Inventors
Paul J. Maddon
Richard Axel M.D.
Manager
Joan Martinez
Departments
Biochemistry & Molecular Biophysics
Divisions
Columbia University Medical Center (CUMC)
Release Date
2024-05-29
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
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
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
Gene
silencing for the treatment of metabolic dysfunction-associated steatohepatitis (MASH)
CU23021
Silencing CASP8-METRN halts progression from steatosis to MASH, offering non-invasive therapy and diagnostic biomarkers.
Tags
Biomarker
Blood plasma
Caspase 8
Inventors
Danish Saleheen
Ira Tabas MD, PhD
Shareef Khalid
Manager
Joan Martinez
Departments
Medicine
Divisions
Columbia University Medical Center (CUMC)
Release Date
2024-11-08
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
