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Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells.
Drouet V, Ruiz M, Zala D, Feyeux M, Auregan G, Cambon K, Troquier L, Carpentier J, Aubert S, Merienne N, Bourgois-Rocha F, Hassig R, Rey M, Dufour N, Saudou F, Perrier AL, Hantraye P, Déglon N. Drouet V, et al. Among authors: deglon n. PLoS One. 2014 Jun 13;9(6):e99341. doi: 10.1371/journal.pone.0099341. eCollection 2014. PLoS One. 2014. PMID: 24926995 Free PMC article.
Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study.
Bloch J, Bachoud-Lévi AC, Déglon N, Lefaucheur JP, Winkel L, Palfi S, Nguyen JP, Bourdet C, Gaura V, Remy P, Brugières P, Boisse MF, Baudic S, Cesaro P, Hantraye P, Aebischer P, Peschanski M. Bloch J, et al. Among authors: deglon n. Hum Gene Ther. 2004 Oct;15(10):968-75. doi: 10.1089/hum.2004.15.968. Hum Gene Ther. 2004. PMID: 15585112 Clinical Trial.
165 results