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Page 1
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: van helsdingen y. 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.
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 helsdingen y. 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.
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: van helsdingen y. J Neuromuscul Dis. 2015;2(s1):S64. J Neuromuscul Dis. 2015. PMID: 27858653 No abstract available.
Fanconi anemia gene mutations are not involved in sporadic Wilms tumor.
Adank MA, Segers H, van Mil SE, van Helsdingen YM, Ameziane N, van den Ouweland AM, Wagner A, Meijers-Heijboer H, Kool M, de Kraker J, Waisfisz Q, van den Heuvel-Eibrink MM. Adank MA, et al. Among authors: van helsdingen ym. Pediatr Blood Cancer. 2010 Oct;55(4):742-4. doi: 10.1002/pbc.22588. Pediatr Blood Cancer. 2010. PMID: 20589654