Amyotrophic lateral sclerosis has not responded well in clinical trials to what initially seemed a promising therapy—the administration of neuronal growth and survival factors. Now, a gene therapy-based approach in mice revives hope that proper delivery of such factors can slow the disease's course.
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Miller, T., Cleveland, D. Has gene therapy for ALS arrived?. Nat Med 9, 1256–1257 (2003). https://doi.org/10.1038/nm1003-1256
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DOI: https://doi.org/10.1038/nm1003-1256
