Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice

Patient samples

This study was approved by the Bloomsbury Ethics Committee (no. 08H071382). CD34 + HSC were obtained from the bone marrow of a 3-month-old severe DADA2 patient presenting with Pure Red-Cell Aplasia (PRCA) (genotype ADA2 p.G47W/p.G47W). Healthy donor HSC were obtained from leukapheresis provided from the Antony Nolan laboratories with consent (no. CGT125, 2 healthy donors: age 21 and 31 years old, one male, one female).

Mice

NBSGW mice were obtained from the Jackson Laboratory. Procedures were approved by the UCL Biological…