Search Results - caroline+kuo

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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 | 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

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