Gene therapy prevents disease and death from non-ketotic hyperglycinemia
Alejandro Lopez‐Ramirez, Adviti Bali, Md. Suhail Alam†, Prasad Kottayil Padmanabhan, Shaun Calhoun, Caroline Bickerton, Ana Lidia Flores‐Mireles, Kasturi Haldar
This study demonstrates that gene therapy using rAAV9-GLDC prevents disease and death in a mouse model of non-ketotic hyperglycinemia.
bioRxiv · Mar 26, 2025 · PreprintNeuroscience