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

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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: 11/20/2024 | 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: 8/14/2024 | 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: 4/12/2024 | 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: 3/8/2024 | 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: 3/6/2024 | 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: 9/15/2023 | Inventor(s): David Williams

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

Category(s): Therapeutics > Gene Therapy And Editing

AAV-Based Treatment of Guanidinoacetate Methyltransferase (GAMT) Deficiency (UCLA Case No. 2021-012)

SUMMARY: UCLA researchers in the Departments Molecular & Medical Pharmacology and Surgery in the David Geffen School of Medicine have developed a novel and tissue-specific gene therapy treatment for Guanidinoacetate methyltransferase (GAMT) deficiency. BACKGROUND: Guanidinoacetate Methyltransferase (GAMT) deficiency is caused by a mutation in...

Published: 8/10/2023 | Inventor(s): Gerald Lipshutz

Keywords(s): Adeno-associated viruses (AAV), Creatine, GAMT deficiency, Gene Therapy, Metabolic Diseases

Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Genetic Diseases, Therapeutics > Metabolism And Endocrinology

2020-152 Method to Clone Human T Cell Receptors (TCRs) from Single Lymphocytes Based on Functional Profiling

SUMMARY: UCLA researchers from the Department of Microbiology, Immunology, and Molecular Genetics have developed a novel method to clone human T cell receptors from single lymphocytes. BACKGROUND: Immunotherapy is a growing therapy type for the treatment of disease, especially cancer, by activating or repressing the immune system. One major type...

Published: 11/15/2024 | Inventor(s): Owen Witte, Jami Witte, Pavlo Nesterenko

Keywords(s):

Category(s): Life Science Research Tools, Therapeutics, Therapeutics > Autoimmune, Therapeutics > Oncology, Therapeutics > Immunology And Immunotherapy, Therapeutics > Gene Therapy And Editing

2022-178: CRISPR-MEDIATED DELETION OF FLI1 IN NK CELLS

SUMMARY: UCLA researchers from the Department of Microbiology, Immunology, and Molecular Genetics have developed a novel method to increase the immune response of natural killer cells by ablating the transcriptional regulator Fli1 via CRISPR. BACKGROUND: Natural killer (NK) cells are innate immune cells that combat viral infections and serve as the...

Published: 9/5/2023 | Inventor(s): Timothy O'Sullivan

Keywords(s):

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

2020-889 293T PACKAGING CELLS WITH TARGETED GENE KNOCKOUTS THAT IMPROVE LENTIVIRAL VECTOR TITERS

INTRODUCTIONUCLA researchers in the Department of Microbiology, Immunology, & Molecular Genetics have found that the knockout of four host cell antiviral factors could increase lentiviral titers, allowing the potential to reduce common off target toxicities of gene therapy while lowering the cost of treatment manufacturing. BACKGROUNDGene therapy...

Published: 11/15/2024 | Inventor(s): Donald Kohn

Keywords(s):

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

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