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Review
. 2012 Nov;48(2):236-42.
doi: 10.1016/j.nbd.2011.08.018. Epub 2011 Aug 25.

Gene therapy for amyotrophic lateral sclerosis

Thais Federici  1 , Nicholas M Boulis
Affiliations
Review

Gene therapy for amyotrophic lateral sclerosis

Thais Federici et al. Neurobiol Dis. 2012 Nov.

Abstract

Gene therapy continues to be a potential option for amyotrophic lateral sclerosis (ALS). This chapter will inform the reader about promising therapeutic transgenes and proof-of-principle studies in transgenic rodent models of ALS. Challenges regarding the disease targets and time for therapeutic intervention will be also discussed. Finally, restorative therapy for ALS, as well as gene therapy for other motor neuron diseases will be briefly reviewed.

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