Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug

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1. 1In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells2015 · 181 citations
2. 2The Role of Conditioning in Hematopoietic Stem-Cell Gene Therapy2016 · 56 citations
3. 3Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)–deficient mice and corrects their immune and metabolic defects2006 · 77 citations
4. 4Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans2012 · 236 citations
5. 5Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning2006 · 219 citations