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Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G. Huston MW, et al. Among authors: blundell mp. Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12. Mol Ther. 2011. PMID: 21750532 Free PMC article.
Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment.
Sabri S, Foudi A, Boukour S, Franc B, Charrier S, Jandrot-Perrus M, Farndale RW, Jalil A, Blundell MP, Cramer EM, Louache F, Debili N, Thrasher AJ, Vainchenker W. Sabri S, et al. Among authors: blundell mp. Blood. 2006 Jul 1;108(1):134-40. doi: 10.1182/blood-2005-03-1219. Epub 2006 Mar 7. Blood. 2006. PMID: 16522820 Free article.