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Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro.
Exp Hematol. 2006 Sep;34(9):1161-9. doi: 10.1016/j.exphem.2006.04.021.
Exp Hematol. 2006.
PMID: 16939809
Free article.
Clinical Trial.
Multiple independent second-site mutations in two siblings with somatic mosaicism for Wiskott-Aldrich syndrome.
Boztug K, Germeshausen M, Avedillo Díez I, Gulacsy V, Diestelhorst J, Ballmaier M, Welte K, Maródi L, Chernyshova L, Klein C.
Boztug K, et al. Among authors: avedillo diez i.
Clin Genet. 2008 Jul;74(1):68-74. doi: 10.1111/j.1399-0004.2008.01019.x. Epub 2008 May 13.
Clin Genet. 2008.
PMID: 18479478
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Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy.
Avedillo Díez I, Zychlinski D, Coci EG, Galla M, Modlich U, Dewey RA, Schwarzer A, Maetzig T, Mpofu N, Jaeckel E, Boztug K, Baum C, Klein C, Schambach A.
Avedillo Díez I, et al.
Mol Pharm. 2011 Oct 3;8(5):1525-37. doi: 10.1021/mp200132u. Epub 2011 Aug 31.
Mol Pharm. 2011.
PMID: 21851067
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