Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease.

Autor: Han SO; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA., Gheorghiu D; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA., Chang A; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA., Mapatano SH; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA., Li S; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA., Brooks E; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA.; Department of Laboratory Animal Resources, Duke University, Durham, North Carolina, USA., Koeberl D; Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA.; Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA.

Publikováno v: Human gene therapy [Hum Gene Ther] 2022 May; Vol. 33 (9-10), pp. 479-491. Date of Electronic Publication: 2022 May 04.