Efficient delivery of FMR1 across the blood brain barrier using 2 AAVphp construct in adult FMR1 KO mice demonstrates the 3 feasibility of gene therapy for fragile X syndrome
Kathryn K. Chadman, Tatyana Adayev, Aishwarya Udayan, Rida Ahmed, Chun‐Ling Dai, Jeffrey H. Goodman, Harry C. Meeker, Natalia Dolzhanskaya, Milen T. Velinov
This study demonstrates efficient brain-specific delivery of FMR1 via AAVphp vectors in adult FMR1 KO mice, leading to partial behavioral improvements and increased FMRP levels.
bioRxiv · Dec 22, 2022 · PreprintGenetics