Injection of novel gene therapy vector prolonged survival in mouse model of Pompe disease

(Mary Ann Liebert, Inc./Genetic Engineering News) A new study has shown that a single injection of a novel adeno-associated vector (AAV)-based therapy can result in improved enzyme activity and glycogen clearance as well as prolonged survival in a mouse model of Pompe disease.

Source: EurekAlert! - Medicine and Health - August 13, 2018 Category: International Medicine & Public Health Source Type: news