학술논문
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.
Document Type
Article
Author
Suk See De Ravin; Xiaolin Wu; Moir, Susan; Kardava, Lela; Anaya-O’Brien, Sandra; Kwatemaa, Nana; Littel, Patricia; Theobald, Narda; Uimook Choi; Ling Su; Marquesen, Martha; Hilligoss, Dianne; Lee, Janet; Buckner, Clarissa M.; Zarember, Kol A.; O’Connor, Geraldine; McVicar, Daniel; Kuhns, Douglas; Throm, Robert E.; Sheng Zhou
Source
Science Translational Medicine; 4/20/2016, Vol. 8 Issue 335, p1-11, 11p, 1 Color Photograph, 5 Graphs
Subject
Language
ISSN
19466234
Abstract
The article discusses the use of a lentiviral gene therapy approach for the treatment of X-linked severe combined immunodeficiency (SCID-X1) patients with persistent immune dysfunction despite haploidentical hematopoietic stem cell (HSC) transplant in infancy. The achievement of similar gene-marking levels in three younger patients is explored. The capability of the therapy to restore humoral immune function to posthaploidentical transplant older patients with SCID-X1 is also tackled.