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131 results
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alpha-L-Iduronidase transport in neurites.
Chen F, Vitry S, Hocquemiller M, Desmaris N, Ausseil J, Heard JM. Chen F, et al. Among authors: heard jm. Mol Genet Metab. 2006 Apr;87(4):349-58. doi: 10.1016/j.ymgme.2005.11.017. Epub 2006 Jan 24. Mol Genet Metab. 2006. PMID: 16439176
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial.
Tardieu M, Zérah M, Gougeon ML, Ausseil J, de Bournonville S, Husson B, Zafeiriou D, Parenti G, Bourget P, Poirier B, Furlan V, Artaud C, Baugnon T, Roujeau T, Crystal RG, Meyer C, Deiva K, Heard JM. Tardieu M, et al. Among authors: heard jm. Lancet Neurol. 2017 Sep;16(9):712-720. doi: 10.1016/S1474-4422(17)30169-2. Epub 2017 Jul 14. Lancet Neurol. 2017. PMID: 28713035 Clinical Trial.
Lack of an immune response against the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated transgene expression in nonhuman primates after intramuscular injection of recombinant adeno-associated virus.
Favre D, Blouin V, Provost N, Spisek R, Porrot F, Bohl D, Marmé F, Chérel Y, Salvetti A, Hurtrel B, Heard JM, Rivière Y, Moullier P. Favre D, et al. Among authors: heard jm. J Virol. 2002 Nov;76(22):11605-11. doi: 10.1128/jvi.76.22.11605-11611.2002. J Virol. 2002. PMID: 12388721 Free PMC article.
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: heard jm. 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.
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: heard jm. 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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