Search Results - gene+therapy

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Soft Bio-Integrated Catheter System
NU 2020-150 INVENTORS John Rogers (Northwestern University, Department of Materials Science and Engineering)* Igor Efimov (Northwestern University, Department of Biomedical Engineering, Medicine)* Roozbeh Ghaffari (Northwestern University, Department of Biomedical Engineering)* Mengdi Han SHORT DESCRIPTION This novel catheter platform delivers...
Published: 12/9/2025   |   Inventor(s):  
Keywords(s): Gene therapy, Sensors
Category(s): Life Sciences > Healthcare Devices, Tools & IT
Human Artificial Chromosome Generation Technology
Technology allowing efficient formation, delivery, isolation, and enrichment of single-copy Human Artificial Chromosome (HAC) for use in research, plant production, gene and cell therapy and more. Problem: Existing gene-editing techniques have limitations due to their reliance on viruses for delivery, restricting their packaging capacities. A more potent...
Published: 9/19/2025   |   Inventor(s): Ben Black, Craig Gambogi, John Glass, David Brown, Gabriel Birchak
Keywords(s): Bioengineering, Gene Editing, Gene Therapy, Genetic Disorders, Platform Technology
Category(s): Technology Classifications > Research Tools & Reagents, Technology Classifications > Therapeutics
TYRP-1 Chimeric Antigen Receptor to Treat Melanoma (UCLA Case No. 2020-106)
UCLA researchers in the Departments of Surgical Oncology and Microbiology, Immunology, & Molecular Genetics. have developed a CAR-T therapy that targets TYRP-1 for the treatment of melanoma. BACKGROUND: Immune checkpoint blockade (ICB) is a rapidly emerging immunotherapy approach that uses patients’ immune cells to treat cancer. In contrast...
Published: 9/15/2025   |   Inventor(s): Cristina Puig Saus, Antoni Ribas, Yvonne Chen
Keywords(s): Gene Therapy, Hematology, Immunotherapy, Research Methods
Category(s): Therapeutics > Hematology
Cell-type specific disruption of M1 muscarinic acetylcholine receptor expression to alleviate Parkinson's disease
NU 2024-244 INVENTORS Dalton J. Surmeier Jr* Tatiana Tkatch Shenyu Zhai SHORT DESCRIPTION Gene therapy targeting M1 muscarinic receptor expression in striatal spiny projection neurons to enhance levodopa response and slow Parkinson’s Disease progression. BACKGROUND Parkinson’s Disease (PD) is the second most common age-related...
Published: 1/5/2026   |   Inventor(s):  
Keywords(s): CNS - Central Nervous System, Gene therapy, Neurodegenerative disease, Neurologic disease, Neurology, PD - Parkinson's Disease, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
Biocompatible And Low-Toxicity Lipid Nanoparticles (LNPs) For Selectively Delivering mRNA To The Bone Microenvironment
Ionizable LNPs with a rigid backbone of piperazine (PIP) linked to bisphosphonates (BPs) for the targeted delivery of mRNA to the bone. Problem: Available treatments for bone tissue related diseases provide only symptomatic relief and are associated with clinical challenges and potential side effects. Challenges persist in the delivery of therapeutic...
Published: 10/3/2025   |   Inventor(s): Michael Mitchell, Kyle Vining, Lulu Xue, Il-Chul Yoon
Keywords(s): Aging, Bioengineering, Drug Delivery, Gene Therapy, Orthopedics, Platform Technology, Women's Health
Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Therapeutics
Viral Vector-Based Gene Therapy for OPA1 Gene Mutation-Induced Dominant Optic Atrophy (UCLA Case No. 2025-004)
UCLA researchers in the Department of Ophthalmology have developed a viral vector-based gene therapy to restore OPA1 protein levels in patients with OPA1-mutated dominant optic atrophy. The optimized vectors will be delivered intravitreally to patients’ eyes, providing a minimally invasive, effective treatment with reduced side effects. BACKGROUND:...
Published: 11/25/2025   |   Inventor(s): Xianjie Yang
Keywords(s): Adeno-associated viruses (AAV), disease model, dominant optic atrophy, Gene Therapy, intravitreal delivery, Lentivirus Viral Vector, mitochondria, mutation, Neuron, OPA1, Ophthalmology, pluripotent stem cell (PSC), retinal ganglion cell
Category(s): Therapeutics > CNS and Neurology, Therapeutics > Gene Therapy And Editing, Therapeutics > Ophthalmology
Recombinant Adeno-Associated Virus 8 Human Solute Carrier Family 4 Member 11 (AAV8-hSLC4A11) for Congenital Hereditary Endothelial Dystrophy (CHED) (UCLA Case No. 2025-096)
UCLA researchers from the Department of Ophthalmology have developed a novel gene therapy method to treat Congenital Hereditary Endothelial Dystrophy. BACKGROUND: Congenital Hereditary Endothelial Dystrophy (CHED) is a rare autosomal recessive disease that affects corneal endothelial cells, which can lead to blindness if left untreated. CHED is more...
Published: 10/13/2025   |   Inventor(s): Anthony Aldave
Keywords(s): Gene Therapy
Category(s): Therapeutics > Ophthalmology, Therapeutics > Gene Therapy And Editing
Gene Therapy for DOCK8 Deficiency (UCLA Case No. 2023-178)
UCLA researchers in the Department of Pediatrics Allergy and Immunology have developed a gene therapy method to express the full-length DOCK8 protein in cells, aimed at treating DOCK8 deficiency. These method allows for safe and effective DOCK8 expression in hematopoietic stem cells without the need for an HLA-matched donor, enabling patients to undergo...
Published: 7/17/2025   |   Inventor(s): Caroline Kuo
Keywords(s): bone marrow, bone marrow transplants, DOCK8, DOCK8 deficiency, Gene Editing Systems, Gene Therapy, hematopoietic stem cells, Immune System, immunodeficiency, Immunology, intein splicing
Category(s): Therapeutics > Immunology And Immunotherapy, Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Life Science Research Tools > Plasmids/Vectors
KIBRA-PKMzeta Interactions in LTP and Memory Maintenance
This technology offers the potential for developing novel and effective treatments for mood- and memory-related disorders and neuropathic pain. Background: During learning, neurons produce an enzyme called protein kinase M zeta (PKMzeta) which is responsible for (1) maintaining newly strengthened connections between neurons, and (2) memory. PKMzeta...
Published: 12/4/2025   |   Inventor(s): Todd Sacktor
Keywords(s): gene therapy, Technologies
Category(s): Campus > SUNY Downstate Health Sciences University, Technology Classifications > Drug Delivery, Technology Classifications > Healthcare, Technology Classifications > Therapeutics and Vaccines
A Method for Promoting Structural and Functional Rescue of Photoreceptors in the Eye to Treat Advanced Retinal Degeneration.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease. Problem: Despite the fact that retinal gene therapy studies have shown promising results in animal models, treatment of advanced retinal degeneration with currently available gene therapies have not been successful....
Published: 9/8/2025   |   Inventor(s): Raghavi Sudharsan, William Beltran
Keywords(s): Bioengineering, Gene Therapy, Genetic Disorders, Ophthalmology, Orphan Disease, Platform Technology, Regenerative Medicine
Category(s): Technology Classifications > Therapeutics
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