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Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease.
Stok M, de Boer H, Huston MW, Jacobs EH, Roovers O, Visser TP, Jahr H, Duncker DJ, van Deel ED, Reuser AJJ, van Til NP, Wagemaker G. Stok M, et al. Among authors: huston mw. Mol Ther Methods Clin Dev. 2020 May 4;17:1014-1025. doi: 10.1016/j.omtm.2020.04.023. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32462050 Free PMC article.
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. 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.
Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE.
Yadak R, Cabrera-Pérez R, Torres-Torronteras J, Bugiani M, Haeck JC, Huston MW, Bogaerts E, Goffart S, Jacobs EH, Stok M, Leonardelli L, Biasco L, Verdijk RM, Bernsen MR, Ruijter G, Martí R, Wagemaker G, van Til NP, de Coo IFM. Yadak R, et al. Among authors: huston mw. Mol Ther Methods Clin Dev. 2018 Jan 8;8:152-165. doi: 10.1016/j.omtm.2018.01.001. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2018. PMID: 29687034 Free PMC article.
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Yasuda M, Huston MW, Pagant S, Gan L, St Martin S, Sproul S, Richards D, Ballaron S, Hettini K, Ledeboer A, Falese L, Cao L, Lu Y, Holmes MC, Meyer K, Desnick RJ, Wechsler T. Yasuda M, et al. Among authors: huston mw. Mol Ther Methods Clin Dev. 2020 Jul 9;18:607-619. doi: 10.1016/j.omtm.2020.07.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775495 Free PMC article.
Epigenetic control of multiple genes with a lentiviral vector encoding transcriptional repressors fused to compact zinc finger arrays.
Monteferrario D, David M, Tadi SK, Zhou Y, Marchetti I, Jeanneau C, Saviane G, Dupont CF, Martelli AE, Truong LN, Eshleman JA, Ng CC, Huston MW, Davis GD, Fontenot JD, Reik A, Rosa M, Fenard D. Monteferrario D, et al. Among authors: huston mw. Mol Ther Methods Clin Dev. 2024 Apr 24;32(2):101255. doi: 10.1016/j.omtm.2024.101255. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38715734 Free PMC article.