Search Results - gene+therapy

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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: 11/7/2025 | 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

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: 5/13/2025 | 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

CHST6 Mini Gene - CHST6 Expression Construct With Native Promoter (UCLA Case No. 2023-301)

UCLA researchers from the Department of Human Genetics and Department of Ophthalmology have developed a mini gene encoding a protein which is mutated in macular corneal dystrophy, leading to a potential gene therapy for this disorder. BACKGROUND: Macular corneal dystrophy (MCD) is a rare genetic disease caused by mutations in the CHST6 gene, which...

Published: 7/17/2025 | Inventor(s): Wenlin Zhang, Anthony Aldave

Keywords(s): AAV, Adeno-associated viruses (AAV), Gene Editing Systems, Gene Therapy, genetics, keratinocytes, macular corneal dystrophy, mini gene, mutation, native promoter, Ophthalmology

Category(s): Therapeutics > Ophthalmology, Therapeutics > Gene Therapy And Editing

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