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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   |   Inventor(s): Donald Kohn, Paul Ayoub
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine