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

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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

A Potent Short Hairpin RNA (SHRNA734) to Positively Select and Eliminate Genetically Modified Cells (UCLA Case No. 2016-524)

BACKGROUND: Gene therapy strategies to modify human stem cells hold great promise for curing many human diseases. However, previous clinical studies have met with limited success, largely due to the low engraftment of gene modified stem cells. One strategy to overcome this challenge involves engineering stem cells in which HPRT expression is knocked...

Published: 3/6/2025 | Inventor(s): Dong Sung An, Saki Shimizu

Keywords(s):

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

Inhibition of BMI1 Eliminated Cancer Stem Cells and Activates Antitumor Immunity (UCLA Case No. 2020-858)

UCLA researchers in the Department of Dentistry discovered that targeting BMI1in head and neck squamous cell carcinoma (HNSCC) may be a viable immune checkpoint to inhibit metastatic tumor growth, and prevent tumor relapse through activation of cell-intrinsic immunity and purging cancer stem cells (CSCs). BACKGROUND: Cancer stem cells are cancer...

Published: 2/14/2025 | Inventor(s): Cun Yu Wang, Jia Lingfei

Keywords(s): Cancer stem cells, Epigenetics, Immunooncology, Oncology Immunotherapy, Tumor

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

Gene Augmentation Therapy for Usher Syndrome 1B (UCLA Case No. 2023-138)

UCLA researchers in the Department of Ophthalmology have developed a novel approach to cure Usher Syndrome 1B. BACKGROUND: Usher syndrome is defined as a condition whose major symptoms are deafness or hearing loss, and an eye disease called retinitis pigmentosa (RP), which affects vision. The ear-related effects of Usher’s disease are caused...

Published: 2/14/2025 | Inventor(s): David Williams

Keywords(s): Gene delivery, Genetic Disease, Hearing loss, Usher syndrome, Vision loss

Category(s): Therapeutics > Gene Therapy And Editing

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