50
Participants
Start Date
January 31, 2015
Primary Completion Date
October 31, 2025
Study Completion Date
October 31, 2025
transduced CD34+ hematopoietic stem cell
Autologous hematopoietic stem cells (HSCs) collected from the mobilized peripheral blood and transduced ex vivo with a Lentiviral vector encoding the human ARSA(for MLD)/ABCD1(for ALD) cDNA（complementary DNA）. Dose: ≥ 2x10\^6 transduced CD34+ cells/Kg (maximum 20x10\^6) at bedside for infusion.
RECRUITING
Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen
Shenzhen University
OTHER
Guangzhou Women and Children's Medical Center
OTHER
Shenzhen Second People's Hospital
OTHER