NeurosciencebioRxivPreprint
Gene therapy prevents disease and death from non-ketotic hyperglycinemia
Alejandro Lopez‐Ramirez, Adviti Bali, Md. Suhail Alam†…·Kasturi Haldar
This study demonstrates that gene therapy using rAAV9-GLDC prevents disease and death in a mouse model of non-ketotic hyperglycinemia.
2 citations