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Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy.
Mol Pharm. 2011 Oct 3;8(5):1525-37. doi: 10.1021/mp200132u. Epub 2011 Aug 31.
Mol Pharm. 2011.
PMID: 21851067
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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Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro.
Dewey RA, Avedillo Díez I, Ballmaier M, Filipovich A, Greil J, Güngör T, Happel C, Maschan A, Noyan F, Pannicke U, Schwarz K, Snapper S, Welte K, Klein C.
Dewey RA, et al. Among authors: avedillo diez i.
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.
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