RT Journal
A1 Stephenson J
T1 GEne therapy and ald
JF JAMA
JO JAMA
YR 2009
FD December 16
VO 302
IS 23
SP 2531
OP 2531
DO 10.1001/jama.2009.1841
UL http://dx.doi.org/10.1001/jama.2009.1841
AB 
ADL, which is caused by mutations in the gene that encodes the ALD protein, can be treated with hematopoietic stem cell transplantation, but the disorder is fatal when a suitable matched donor is not available. The new approach involves removing blood stem cells from the patient and using a lentiviral vector derived from a disabled version of HIV to introduce a working copy of the ALD gene into these cells. The patient then receives an infusion of the modified cells after undergoing myeloablative treatment to destroy the bone marrow.