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Page 1
Gene Therapy Developments for Pompe Disease.
Unnisa Z, Yoon JK, Schindler JW, Mason C, van Til NP. Unnisa Z, et al. Among authors: van til np. Biomedicines. 2022 Jan 28;10(2):302. doi: 10.3390/biomedicines10020302. Biomedicines. 2022. PMID: 35203513 Free PMC article. Review.
High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing.
Plasschaert RN, DeAndrade MP, Hull F, Nguyen Q, Peterson T, Yan A, Loperfido M, Baricordi C, Barbarossa L, Yoon JK, Dogan Y, Unnisa Z, Schindler JW, van Til NP, Biasco L, Mason C. Plasschaert RN, et al. Among authors: van til np. Mol Ther. 2022 Oct 5;30(10):3209-3225. doi: 10.1016/j.ymthe.2022.05.022. Epub 2022 May 25. Mol Ther. 2022. PMID: 35614857 Free PMC article.
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease.
Dogan Y, Barese CN, Schindler JW, Yoon JK, Unnisa Z, Guda S, Jacobs ME, Oborski C, Maiwald T, Clarke DL, Schambach A, Pfeifer R, Harper C, Mason C, van Til NP. Dogan Y, et al. Among authors: van til np. Mol Ther Methods Clin Dev. 2022 Nov 3;27:464-487. doi: 10.1016/j.omtm.2022.10.017. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36419467 Free PMC article.
Gene and Cellular Therapies for Leukodystrophies.
Aerts-Kaya F, van Til NP. Aerts-Kaya F, et al. Among authors: van til np. Pharmaceutics. 2023 Oct 24;15(11):2522. doi: 10.3390/pharmaceutics15112522. Pharmaceutics. 2023. PMID: 38004502 Free PMC article. Review.
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: van der wegen p, van der ploeg at. 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.
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: van til np. 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 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: van til np, van deel ed. 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 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: van til np. 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.
40 results