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Current developments in gene therapy for amyotrophic lateral sclerosis

10 May 2015

Abstract excerpt

INTRODUCTION: Amyotrophic lateral sclerosis (ALS) is a devastating adult neurodegenerative disorder characterized by motor neuron degeneration and death around 3 years from onset. So far, riluzole is the only treatment available, although it only offers a slight increase in survival. The complex etiology of ALS, with several genes able to trigger the disease, makes its study difficult. AREAS COVERED: RNA-mediated...

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PMID: 25959569
DOI: 10.1517/14712598.2015.1044894

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