GeneticsbioRxivPreprint
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…·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.
DOI 10.1101/2022.12.22.520936