Globin gene therapy: one (seemingly) small vector change, one giant leap in optimism.
Bodine
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National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, MSC 4442, Bethesda, Maryland 20892-4442, USA. tedyaz@nchgr.nih.gov
D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005914:Globins / Q000235:genetics*; D015497:HIV-1 / Q000235:genetics; D006453:Hemoglobinopathies / Q000628:therapy; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D051379:Mice; D012333:RNA, Messenger / Q000378:metabolism; D014161:Transduction, Genetic
D012333:RNA, Messenger; D005914:Globins