Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system
Jennifer L. Watts, Shibi B. Likhite, Yoshiko Murakami, Taroh Kinoshita, Kathrin Meyer, Rolf W. Stottmann
This study uses AAV9-mediated PIGA gene replacement to rescue PIGA deficiency phenotypes in mice, improving survival, brain structure, and neurological function.
bioRxiv · Dec 13, 2025 · PreprintGenetics