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Search Results - therapeutics+%3e+gene+therapy+and+editing
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In Cellulo Synthesis of mRNA-Specific Stoichiometrically-Precise Virus-Like Particles (UCLA Case No. 2024-056)
UCLA researchers from the Department of Chemistry and Biochemistry have developed a novel virus-like particle (VLP) platform for the encapsulation of mRNA for efficient therapeutic delivery. BACKGROUND: Messenger RNA (mRNA) technologies have emerged as powerful therapeutics in vaccines and gene therapies. While traditional methods of mRNA therapeutics...
Published: 12/8/2025
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Inventor(s):
William Gelbart
Keywords(s):
Category(s):
Therapeutics > Vaccines
,
Platforms > Drug Delivery
,
Therapeutics > Gene Therapy And Editing
,
Materials > Nanotechnology
Nanoparticle & dsDNA Toolkit for Decreased Toxicity and Gene Editing of Epithelial Stem Cells via Epithelial Disruption (UCLA Case No. 2023-205/209) -- for Cystic Fibrosis and other genetic diseases
UCLA researchers in the Department of Pediatrics, Hematology/ Oncology have constructed a nanoparticle toolkit for increased integration and reduced toxicity for gene therapies that treat debilitating diseases such as Cystic Fibrosis and other critical genetic diseases. BACKGROUND: Gene therapies are the forefront of therapeutic advancements as a one-off...
Published: 12/1/2025
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Inventor(s):
Steven Jonas
,
Brigitte Gomperts
,
Donald Kohn
Keywords(s):
Category(s):
Therapeutics > Gene Therapy And Editing
,
Platforms > Drug Delivery
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
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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
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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
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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: 11/12/2025
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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
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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
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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: 11/25/2025
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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
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Inventor(s):
Anthony Aldave
Keywords(s):
Gene Therapy
Category(s):
Therapeutics > Ophthalmology
,
Therapeutics > Gene Therapy And Editing
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