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C9ORF72 hexanucleotide repeat exerts toxicity in a stable, inducible motor neuronal cell model, which is rescued by partial depletion of Pten.
Stopford MJ, Higginbottom A, Hautbergue GM, Cooper-Knock J, Mulcahy PJ, De Vos KJ, Renton AE, Pliner H, Calvo A, Chio A, Traynor BJ, Azzouz M, Heath PR; ITALSGEN Consortium, NeuroX Consortium; Kirby J, Shaw PJ. Stopford MJ, et al. Among authors: azzouz m. Hum Mol Genet. 2017 Mar 15;26(6):1133-1145. doi: 10.1093/hmg/ddx022. Hum Mol Genet. 2017. PMID: 28158451 Free PMC article.
Gene therapy: a promising approach to treating spinal muscular atrophy.
Mulcahy PJ, Iremonger K, Karyka E, Herranz-Martín S, Shum KT, Tam JK, Azzouz M. Mulcahy PJ, et al. Among authors: azzouz m. Hum Gene Ther. 2014 Jul;25(7):575-86. doi: 10.1089/hum.2013.186. Epub 2014 Jun 27. Hum Gene Ther. 2014. PMID: 24845847 Review.
201 results