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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. Among authors: petry h. 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.
AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
Evers MM, Miniarikova J, Juhas S, Vallès A, Bohuslavova B, Juhasova J, Skalnikova HK, Vodicka P, Valekova I, Brouwers C, Blits B, Lubelski J, Kovarova H, Ellederova Z, van Deventer SJ, Petry H, Motlik J, Konstantinova P. Evers MM, et al. Among authors: petry h. Mol Ther. 2018 Sep 5;26(9):2163-2177. doi: 10.1016/j.ymthe.2018.06.021. Epub 2018 Jun 25. Mol Ther. 2018. PMID: 30007561 Free PMC article.
Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.
Pañeda A, Lopez-Franco E, Kaeppel C, Unzu C, Gil-Royo AG, D'Avola D, Beattie SG, Olagüe C, Ferrero R, Sampedro A, Mauleon I, Hermening S, Salmon F, Benito A, Gavira JJ, Cornet ME, del Mar Municio M, von Kalle C, Petry H, Prieto J, Schmidt M, Fontanellas A, González-Aseguinolaza G. Pañeda A, et al. Among authors: petry h. Hum Gene Ther. 2013 Dec;24(12):1007-17. doi: 10.1089/hum.2013.166. Hum Gene Ther. 2013. PMID: 24070415
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.
Unzu C, Sampedro A, Mauleón I, Alegre M, Beattie SG, de Salamanca RE, Snapper J, Twisk J, Petry H, González-Aseguinolaza G, Artieda J, Rodríguez-Pena MS, Prieto J, Fontanellas A. Unzu C, et al. Among authors: petry h. Mol Ther. 2011 Feb;19(2):243-50. doi: 10.1038/mt.2010.210. Epub 2010 Sep 28. Mol Ther. 2011. PMID: 20877347 Free PMC article.
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Miniarikova J, Zimmer V, Martier R, Brouwers CC, Pythoud C, Richetin K, Rey M, Lubelski J, Evers MM, van Deventer SJ, Petry H, Déglon N, Konstantinova P. Miniarikova J, et al. Among authors: petry h. Gene Ther. 2017 Oct;24(10):630-639. doi: 10.1038/gt.2017.71. Epub 2017 Aug 3. Gene Ther. 2017. PMID: 28771234 Free PMC article.
228 results