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

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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

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: 7/17/2025 | 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: 7/17/2025 | 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: 8/15/2025 | 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

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

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: 7/17/2025 | Inventor(s): Donald Kohn, Roger Hollis, Richard Morgan

Keywords(s):

Category(s): Therapeutics > Gene Therapy And Editing, Life Science Research Tools, Therapeutics > Genetic Diseases

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