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Gene Repair for Hemophilia A (UCLA Case No. 2014-016)
Gene Repair For Hemophilia ASUMMARYResearchers at UCLA from the Departments of Medicine and Urology have developed a treatment for hemophilia A that utilizes non-viral gene editing technologies for ex vivo repair of the mutation in the gene F8.BACKGROUNDHemophilia affects about 400,000 people worldwide, and approximately 80% of these cases are hemophilia...
Published: 7/17/2025   |   Inventor(s): Thomas Howard, Vincent La Terza
Keywords(s): Gene Therapy, Hematology, Therapeutics & Vaccines
Category(s): Therapeutics, Therapeutics > Hematology, Therapeutics > Gene Therapy And Editing