Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.
Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856.
Sci Transl Med. 2016.
PMID: 27099176
Free PMC article.
Clinical Trial.