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Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Bugiani M, Abbink TEM, Edridge AWD, van der Hoek L, Hillen AEJ, van Til NP, Hu-A-Ng GV, Breur M, Aiach K, Drevot P, Hocquemiller M, Laufer R, Wijburg FA, van der Knaap MS. Bugiani M, et al. Among authors: hocquemiller m. Ann Clin Transl Neurol. 2023 Jun;10(6):904-917. doi: 10.1002/acn3.51772. Epub 2023 May 11. Ann Clin Transl Neurol. 2023. PMID: 37165777 Free PMC article.
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. 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.
Design of a stable human acid-β-glucosidase: towards improved Gaucher disease therapy and mutation classification.
Pokorna S, Khersonsky O, Lipsh-Sokolik R, Goldenzweig A, Nielsen R, Ashani Y, Peleg Y, Unger T, Albeck S, Dym O, Tirosh A, Tarayra R, Hocquemiller M, Laufer R, Ben-Dor S, Silman I, Sussman JL, Fleishman SJ, Futerman AH. Pokorna S, et al. Among authors: hocquemiller m. FEBS J. 2023 Jul;290(13):3383-3399. doi: 10.1111/febs.16758. Epub 2023 Mar 23. FEBS J. 2023. PMID: 36808692
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. 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.
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.
Hocquemiller M, Giersch L, Audrain M, Parker S, Cartier N. Hocquemiller M, et al. 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.
Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial.
Tardieu M, Zérah M, Husson B, de Bournonville S, Deiva K, Adamsbaum C, Vincent F, Hocquemiller M, Broissand C, Furlan V, Ballabio A, Fraldi A, Crystal RG, Baugnon T, Roujeau T, Heard JM, Danos O. Tardieu M, et al. Among authors: hocquemiller m. Hum Gene Ther. 2014 Jun;25(6):506-16. doi: 10.1089/hum.2013.238. Epub 2014 May 5. Hum Gene Ther. 2014. PMID: 24524415 Clinical Trial.
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