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Page 1
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. 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.
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype.
van Til NP, Stok M, Aerts Kaya FS, de Waard MC, Farahbakhshian E, Visser TP, Kroos MA, Jacobs EH, Willart MA, van der Wegen P, Scholte BJ, Lambrecht BN, Duncker DJ, van der Ploeg AT, Reuser AJ, Verstegen MM, Wagemaker G. van Til NP, et al. Among authors: stok m. Blood. 2010 Jul 1;115(26):5329-37. doi: 10.1182/blood-2009-11-252874. Epub 2010 Apr 12. Blood. 2010. PMID: 20385789 Free article.
Lentiviral Stem Cell Gene Therapy for Pompe Disease.
Liang Q, Stok M, van Helsdingen Y, van der Velden G, Jacobs E, Duncker D, Reuser A, van der Ploeg A, Vulto A, van Til NP, Wagemaker G. Liang Q, et al. Among authors: stok m. J Neuromuscul Dis. 2015;2(s1):S64. J Neuromuscul Dis. 2015. PMID: 27858653 No abstract available.
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: stok m. 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.
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.
Liang Q, Vlaar EC, Catalano F, Pijnenburg JM, Stok M, van Helsdingen Y, Vulto AG, Unger WWJ, van der Ploeg AT, Pijnappel WWMP, van Til NP. Liang Q, et al. Among authors: stok m. Mol Ther Methods Clin Dev. 2022 May 4;25:520-532. doi: 10.1016/j.omtm.2022.04.016. eCollection 2022 Jun 9. Mol Ther Methods Clin Dev. 2022. PMID: 35662813 Free PMC article.
IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy.
Liang Q, Catalano F, Vlaar EC, Pijnenburg JM, Stok M, van Helsdingen Y, Vulto AG, van der Ploeg AT, van Til NP, Pijnappel WWMP. Liang Q, et al. Among authors: stok m. Mol Ther Methods Clin Dev. 2022 Sep 24;27:109-130. doi: 10.1016/j.omtm.2022.09.010. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36284764 Free PMC article.
Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy.
de Vries JM, Kuperus E, Hoogeveen-Westerveld M, Kroos MA, Wens SC, Stok M, van der Beek NA, Kruijshaar ME, Rizopoulos D, van Doorn PA, van der Ploeg AT, Pijnappel WW. de Vries JM, et al. Among authors: stok m. Genet Med. 2017 Jan;19(1):90-97. doi: 10.1038/gim.2016.70. Epub 2016 Jun 30. Genet Med. 2017. PMID: 27362911 Free article.
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
Visigalli I, Delai S, Politi LS, Di Domenico C, Cerri F, Mrak E, D'Isa R, Ungaro D, Stok M, Sanvito F, Mariani E, Staszewsky L, Godi C, Russo I, Cecere F, Del Carro U, Rubinacci A, Brambilla R, Quattrini A, Di Natale P, Ponder K, Naldini L, Biffi A. Visigalli I, et al. Among authors: stok m. Blood. 2010 Dec 9;116(24):5130-9. doi: 10.1182/blood-2010-04-278234. Epub 2010 Sep 16. Blood. 2010. PMID: 20847202 Free PMC article.
Response to Herbert et al.
de Vries JM, Kuperus E, Hoogeveen-Westerveld M, Kroos MA, Wens SCA, Stok M, van der Beek NAME, Kruijshaar ME, Rizopoulos D, van Doorn PA, van der Ploeg AT, Pijnappel WWMP. de Vries JM, et al. Among authors: stok m. Genet Med. 2017 Nov;19(11):1283-1284. doi: 10.1038/gim.2017.49. Epub 2017 Jun 29. Genet Med. 2017. PMID: 28661488 Free article. No abstract available.
41 results