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Gene therapy of the brain in the dog model of Hurler's syndrome.
Ciron C, Desmaris N, Colle MA, Raoul S, Joussemet B, Vérot L, Ausseil J, Froissart R, Roux F, Chérel Y, Ferry N, Lajat Y, Schwartz B, Vanier MT, Maire I, Tardieu M, Moullier P, Heard JM. Ciron C, et al. Among authors: heard jm. Ann Neurol. 2006 Aug;60(2):204-13. doi: 10.1002/ana.20870. Ann Neurol. 2006. PMID: 16718701
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.
[Gene therapy in lysosomal diseases].
Moullier P, Salvetti A, Bohl D, Danos O, Heard JM. Moullier P, et al. Among authors: heard jm. C R Seances Soc Biol Fil. 1996;190(1):45-51. C R Seances Soc Biol Fil. 1996. PMID: 8881268 Review. French.
138 results