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Gene therapy for severe combined immune deficiency.
Qasim W, Gaspar HB, Thrasher AJ. Qasim W, et al. Among authors: thrasher aj. Expert Rev Mol Med. 2004 Jul 2;6(13):1-15. doi: 10.1017/S1462399404007884. Expert Rev Mol Med. 2004. PMID: 15236670 Review.
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.
Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ. Gaspar HB, et al. Among authors: thrasher aj. Mol Ther. 2006 Oct;14(4):505-13. doi: 10.1016/j.ymthe.2006.06.007. Epub 2006 Aug 14. Mol Ther. 2006. PMID: 16905365 Free article.
Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells.
Zhang F, Thornhill SI, Howe SJ, Ulaganathan M, Schambach A, Sinclair J, Kinnon C, Gaspar HB, Antoniou M, Thrasher AJ. Zhang F, et al. Among authors: thrasher aj. Blood. 2007 Sep 1;110(5):1448-57. doi: 10.1182/blood-2006-12-060814. Epub 2007 Apr 24. Blood. 2007. PMID: 17456723 Free PMC article.
Gene therapy for primary immunodeficiencies.
Thrasher AJ. Thrasher AJ. Immunol Allergy Clin North Am. 2008 May;28(2):457-71, xi. doi: 10.1016/j.iac.2008.02.001. Immunol Allergy Clin North Am. 2008. PMID: 18424342 Review.
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.
Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Zhang F, Adams S, Bjorkegren E, Bayford J, Brown L, Davies EG, Veys P, Fairbanks L, Bordon V, Petropoulou T, Kinnon C, Thrasher AJ. Gaspar HB, et al. Among authors: thrasher aj. Sci Transl Med. 2011 Aug 24;3(97):97ra80. doi: 10.1126/scitranslmed.3002716. Sci Transl Med. 2011. PMID: 21865538 Clinical Trial.
Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing.
Knight S, Zhang F, Mueller-Kuller U, Bokhoven M, Gupta A, Broughton T, Sha S, Antoniou MN, Brendel C, Grez M, Thrasher AJ, Collins M, Takeuchi Y. Knight S, et al. Among authors: thrasher aj. J Virol. 2012 Sep;86(17):9088-95. doi: 10.1128/JVI.00485-12. Epub 2012 Jun 13. J Virol. 2012. PMID: 22696657 Free PMC article.
432 results