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The recent success of a viral-vector-based gene treatment in spinal muscular atrophy-promoting success and motor function with an individual intravenous injection-offers a paradigm for such healing input and a platform to create on. Although challenges remain, the newfound optimism mostly stems from advances within the development of viral vectors that may diffusely deliver genetics through the CNS, as well as genome-engineering tools that may adjust infection pathways in many ways that have be

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