Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia

por Zhang, Shengwen, Bastille, Amy, Gordo, Susana, Ramesh, Nikhil, Vora, Jenisha, McCarthy, Elizabeth, Zhang, Xiaohan, Frank, Dylan, Ko, Chih-Wei, Wu, Carmen, Walsh, Noel, Amarwani, Shreya, Liao, Jing, Xiong, Qiang, Drouin, Lauren, Hebben, Matthias, Chiang, Kyle, Chau, B. Nelson
Publicado 2022