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

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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: 6/20/2025   |   Inventor(s): Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Stem Cells And Regenerative Medicine
UCLA CRISPR Licensing Program
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: 6/12/2025   |   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: 4/22/2025   |   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: 3/6/2025   |   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: 2/25/2025   |   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: 2/18/2025   |   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: 2/18/2025   |   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
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: 2/14/2025   |   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
Augmentations to Lentiviral Vectors to Increase Expression (UCLA Case No. 2019-616)
UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin. BACKGROUND: Sickle cell disease (SCD) is one of the...
Published: 2/14/2025   |   Inventor(s): Donald Kohn, Roger Hollis, Richard Morgan
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Life Science Research Tools, Therapeutics > Genetic Diseases
Methods and Compositions for Modification of the HPRT Locus (UCLA Case No. 2013-570)
UCLA researchers have developed a gene editing strategy and methodology based on nuclease-based insertion. 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 implement this technology....
Published: 2/14/2025   |   Inventor(s): Noriyuki Kasahara
Keywords(s): Gene delivery, Gene Editing Systems, Gene Therapy
Category(s): Therapeutics > Gene Therapy And Editing
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