Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency

Investigator(s):
Organization: 
UCLA
NCT Number: 
Disease Name: 
Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (Cryopreserved)
Disease Category: 
Trial Phases: 
Phase 1/2
Trial Status: 
Closed
Trial Contact: 

Kit L Shaw, Ph.D. 
(310) 825-6188 
kshaw@mednet.ucla.edu