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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