Stem-cell gene therapy for the Wiskott-Aldrich syndrome.
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Boztug K, et al. Among authors: diez ia.
N Engl J Med. 2010 Nov 11;363(20):1918-27. doi: 10.1056/NEJMoa1003548.
N Engl J Med. 2010.
PMID: 21067383
Free PMC article.
Clinical Trial.