Liver-Directed Adeno-Associated Virus\u2013Mediated Gene Therapy for Mucopolysaccharidosis Type VI

Brunetti-Pierri N., Ferla R., Ginocchio V. M., Rossi A., Fecarotta S., Romano R., ...More

NEJM Evidence, vol.1, 2022 (Peer-Reviewed Journal)

  • Publication Type: Article / Article
  • Volume: 1
  • Publication Date: 2022
  • Doi Number: 10.1056/evidoa2200052
  • Journal Name: NEJM Evidence
  • Journal Indexes: Other Indexes
  • Hacettepe University Affiliated: Yes