WEBVTT

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Hi, my name is Charlotte Sumner and I'm
a Professor of Neurology and Neuroscience

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at the Johns Hopkins University
School of Medicine.

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I'd like to tell you about
the remarkable success we've had

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in developing gene-targeted treatments

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for the motor neuron disease
spinal muscular atrophy,

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and how such therapeutic platforms

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may enable us to treat
multiple genetic disorders.

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