Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects
Grant Award Details
Grant Type:
Grant Number:
CLIN2-17078
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$14,325,181
Status:
Active
Grant Application Details
Application Title:
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects
Publications
- N Engl J Med (2025): Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency. (PubMed: 41092330)
