Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice. - Hong Y; Burleigh A; Liao A; Yeung J; Bian Y; Sebire N; Ogunbiyi O; Omoyinmi E; Thrasher AJ; Morris E; Brogan PA; Eleftheriou D (Gene therapy, 2025)