Search Results - therapeutics+%3e+gene+therapy+and+editing

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Bioinformatically-Identified Control Elements for Regulated Expression from a Lentiviral Vector to Treat X-Linked Lymphoproliferative Disease (UCLA Case No. 2022-065)
UCLA researchers from the Department of Microbiology, Immunology, and Molecular Genetics and Pediatrics have used a bioinformatics-guided approach to design regulated lentiviral vectors for the treatment of X-linked lymphoproliferative disease type 1. BACKGROUND: X-linked lymphoproliferative disease type 1 (XLP1) is a rare primary immunodeficiency...
Published: 10/17/2025   |   Inventor(s): Donald Kohn, Paul Ayoub
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine
Lipid Nanoparticle-Mediated Delivery of Gene Editing Reagents for Targeted Disruption of Oncogenic Drivers in C-Myc Overexpressing Malignancies (UCLA Case No. 2025-9A9)
Contact UCLA Technology Development Group to learn more.
Published: 9/19/2025   |   Inventor(s): Steven Jonas, Tanya Stoyanova
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Oncology
Vectors Combining Anti-Sickling Beta-AS3-Globin with Anti BCL11A ShRNAmir to Treat Beta-Hemoglobinopathies (UCLA Case No. 2021-190)
UCLA researchers from the Department of Microbiology, Immunology and Molecular Genetics and Pediatrics have developed a potent bi-functional lentiviral expression vector to treat sickle cell disease and beta-thalassemia. BACKGROUND: Beta-hemoglobinopathies, such as sickle cell disease and beta-thalassemia, are genetic disorders characterized by dysfunctional...
Published: 9/11/2025   |   Inventor(s): Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Stem Cells And Regenerative Medicine
Novel AAV Serotypes Derived From a Library Screen (UCLA Case No. 2020-789)
UCLA researchers in the Department of Neurology have produced novel AAV serotypes for a wide range of tissue and cell specific gene delivery. BACKGROUND: Gene therapies are the forefront of therapeutic advancements for debilitating diseases. However, delivery and insertion of the therapeutic transgene are major hurdles that must be solved to effectively...
Published: 7/17/2025   |   Inventor(s): Melissa Spencer
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing
UCLA CRISPR Licensing Program (Multiple Technology Case Numbers)
UCLA CRISPR Licensing Program A major challenge in CRISPR-based gene editing is efficient delivery of the editing system to target cells. UCLA has filed patents on plant viral delivery vectors expressing highly-miniaturized CRISPR systems that may overcome these challenges. The technology is based on recent work from the Jacobsen (UCLA) and Doudna...
Published: 9/12/2025   |   Inventor(s): Steven Jacobsen, Jennifer Doudna
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing
Gene Modifications to Enhance the Function of PSC-Derived T Cells and NK Cells (UCLA Case No. 2023-129)
UCLA researchers in the Department of Medicine have generated novel pluripotent stem cell derived T cells that exhibit improved survival, expansion, and anti-tumor efficacy for use in cell therapy treatments. BACKGROUND: The Food and Drug Administration has approved several chimeric antigen receptor-T (CAR-T) cell therapies since 2017 to combat lymphoma...
Published: 7/31/2025   |   Inventor(s): Christopher Seet
Keywords(s): CAR-T cell therapy, Immunotherapy, pluripotent stem cell (PSC), T-cell engineering
Category(s): Therapeutics > Immunology And Immunotherapy, Therapeutics > Gene Therapy And Editing
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: 7/17/2025   |   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
Synthetic Exosomes (SE) for CNS Delivery of CRISPR for Gene Editing in Brain Disorders (UCLA Case No. 2021-363)
UCLA researchers from the Department of Neurology have developed synthetic exosomes, a novel delivery technology capable of transporting gene-editing tools and therapeutics across the blood-brain barrier, with successful applications in repairing Alzheimer’s-related mutations and delivering proteins, antibodies, and drugs. BACKGROUND: The emergence...
Published: 7/17/2025   |   Inventor(s): Varghese John
Keywords(s): Alzheimers disease, CRISPR, delivery system, exosome, Nanotechnology, nanovesicles, synthetic biology
Category(s): Life Science Research Tools > Microfluidics And Mems, Materials > Nanotechnology, Platforms > Drug Delivery, Therapeutics > Gene Therapy And Editing, Therapeutics > CNS and Neurology
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
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