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

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Gene Editing of Monogenic Disorders in Human Hematopoietic Stem Cells
Case 2018-372Researchers at the UCLA Department of Microbiology, Immunology & Molecular Genetics have developed novel methods to achieve efficient, precise gene integration and effective expression of cDNA cassettes to express normal versions of genes in hematopoietic stem cells.BACKGROUNDHematopoietic stem cells (HSCs) have great therapeutic potential...
Published: 6/3/2026   |   Updated: 6/3/2026   |   Inventor(s): Donald Kohn, Caroline Kuo, Zulema Romero Garcia, Roger Hollis
Keywords(s): Drug Delivery, Gene Editing Systems, Gene Therapy, Life Science Research Tools
Category(s): Platforms > Drug Delivery, Therapeutics > Gene Therapy And Editing, Life Science Research Tools
In Cellulo Syntheses of Targeting-Ligand-Conjugatable, RNA-Specific, Enveloped Virus-Like Particles (UCLA Case No. 2025-159)
Contact UCLA Technology Development Group to learn more. More info available soon.
Published: 6/16/2026   |   Updated: 6/2/2026   |   Inventor(s): William Gelbart
Keywords(s):  
Category(s): Platforms > Drug Delivery, Therapeutics > Gene Therapy And Editing, Life Science Research Tools > Plasmids/Vectors
Engineered Gamma Delta T-Cells and Methods of Making and Using Thereof (UCLA Case No. 2020-913)
UCLA researchers in the Department of Molecular Immunology & Molecular Genetics have developed gamma delta (gd) T cell receptor gene-modified immune cells derived from stem and progenitor cells that enable scalable, off-the-shelf allogeneic cell therapies with broad therapeutic potential. BACKGROUND: Cell-based immunotherapies have demonstrated...
Published: 3/4/2026   |   Updated: 3/4/2026   |   Inventor(s): Lili Yang, Derek Lee, Tyler Halladay
Keywords(s):  
Category(s): Diagnostic Markers > Immunology, Diagnostic Markers > Cancer, Life Science Research Tools > Cell Lines, Therapeutics > Oncology, Therapeutics > CNS and Neurology, Therapeutics > Hematology, Therapeutics > Autoimmune, Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Infectious Diseases
Gene-Modified Conventional Type 1 Dendritic Cells (UCLA Case No. 2026-093)
UCLA researchers in the Departments of Medicine and Pulmonary Disease have recently developed a novel approach for improving the long-term efficacy of anti-PD-1 immunotherapy in non-small cell lung cancer. BACKGROUND: Non-small cell lung cancer (NSCLC) is one of the most common types of lung cancer. Anti-PD-1 immunotherapy is used for treatment despite...
Published: 5/16/2026   |   Updated: 3/4/2026   |   Inventor(s): Bin Liu, Steven Dubinett
Keywords(s):  
Category(s): Therapeutics > Oncology, Therapeutics > Gene Therapy And Editing, Therapeutics > Vaccines
Regulated Gene Expression During Differentiation of Mature T Cells (UCLA Case No. 2022-147)
UCLA researchers from the Department of Pathology & Laboratory Medicine have developed a process to genetically modify stem cells to express transgenes in a regulated, stage-specific manner, avoiding the negative consequences of transgene expression during T cell development. Contact UCLA Technology Development Group for more information.
Published: 6/25/2026   |   Updated: 1/27/2026   |   Inventor(s): Gay Crooks, Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Oncology, Therapeutics > Immunology And Immunotherapy, Therapeutics > Inflammation And Inflammatory Diseases
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   |   Updated: 12/8/2025   |   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   |   Updated: 12/1/2025   |   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   |   Updated: 10/17/2025   |   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   |   Updated: 9/19/2025   |   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   |   Updated: 9/11/2025   |   Inventor(s): Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Stem Cells And Regenerative Medicine
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