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Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.
Hocquemiller M, Giersch L, Audrain M, Parker S, Cartier N. Hocquemiller M, et al. Among authors: giersch l. Hum Gene Ther. 2016 Jul;27(7):478-96. doi: 10.1089/hum.2016.087. Hum Gene Ther. 2016. PMID: 27267688 Free PMC article. Review.
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
Hocquemiller M, Giersch L, Mei X, Gross AL, Randle AN, Gray-Edwards HL, Hudson JA, Todeasa S, Stoica L, Martin DR, Sena-Esteves M, Aiach K, Laufer R. Hocquemiller M, et al. Among authors: giersch l. Mol Ther Methods Clin Dev. 2022 Oct 7;27:281-292. doi: 10.1016/j.omtm.2022.10.004. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36320411 Free PMC article.
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.
Hocquemiller M, Hemsley KM, Douglass ML, Tamang SJ, Neumann D, King BM, Beard H, Trim PJ, Winner LK, Lau AA, Snel MF, Gomila C, Ausseil J, Mei X, Giersch L, Plavsic M, Laufer R. Hocquemiller M, et al. Among authors: giersch l. Mol Ther Methods Clin Dev. 2019 Dec 10;17:174-187. doi: 10.1016/j.omtm.2019.12.001. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2019. PMID: 31909089 Free PMC article.