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Page 1
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
Catalano F, Vlaar EC, Dammou Z, Katsavelis D, Huizer TF, Zundo G, Hoogeveen-Westerveld M, Oussoren E, van den Hout HJMP, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Among authors: vlaar ec. Hum Gene Ther. 2024 Apr;35(7-8):256-268. doi: 10.1089/hum.2023.177. Epub 2024 Feb 2. Hum Gene Ther. 2024. PMID: 38085235 Free PMC article.
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.
Catalano F, Vlaar EC, Katsavelis D, Dammou Z, Huizer TF, van den Bosch JC, Hoogeveen-Westerveld M, van den Hout HJMP, Oussoren E, Ruijter GJG, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Among authors: vlaar ec. Mol Ther Methods Clin Dev. 2023 Nov 2;31:101149. doi: 10.1016/j.omtm.2023.101149. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38033460 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: vlaar ec. 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: vlaar ec. 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.