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2010 1
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2022 0
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Surrogate Cerebrospinal Fluid Biomarkers for Assessing the Efficacy of Gene Therapy in Hurler Syndrome.
Haseloff RF, Trudel S, Birke R, Schümann M, Krause E, Gomila C, Heard JM, Blasig IE, Ausseil J. Haseloff RF, et al. Front Neurol. 2021 May 13;12:640547. doi: 10.3389/fneur.2021.640547. eCollection 2021. Front Neurol. 2021. PMID: 34054689 Free PMC article.
In a previous study, we demonstrated the feasibility, safety, and efficacy of gene therapy by injecting recombinant adeno-associated viral vector serotype (AAV)2/5-IDUA into the brain of a canine model of MPS I. We report on a quantitative proteomic analysis of control …
In a previous study, we demonstrated the feasibility, safety, and efficacy of gene therapy by injecting recombinant adeno-associated viral v …
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.
Ellinwood NM, Ausseil J, Desmaris N, Bigou S, Liu S, Jens JK, Snella EM, Mohammed EE, Thomson CB, Raoul S, Joussemet B, Roux F, Chérel Y, Lajat Y, Piraud M, Benchaouir R, Hermening S, Petry H, Froissart R, Tardieu M, Ciron C, Moullier P, Parkes J, Kline KL, Maire I, Vanier MT, Heard JM, Colle MA. Ellinwood NM, et al. Mol Ther. 2011 Feb;19(2):251-9. doi: 10.1038/mt.2010.265. Epub 2010 Dec 7. Mol Ther. 2011. PMID: 21139569 Free PMC article.
Studies in the mouse model showed that gene therapy providing the missing enzyme alpha-N-acetyl-glucosaminidase to brain cells prevents neurodegeneration and improves behavior. We now document safety and efficacy in affected dogs. Animals received eight deposits of a serot …
Studies in the mouse model showed that gene therapy providing the missing enzyme alpha-N-acetyl-glucosaminidase to brain cells prevents neur …