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Gene Therapy for Inborn Errors of Metabolism: New Approaches

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The first viable mouse model of combined methylmalonic acidemia and homocystinuria, cblC type displays a sustained response to a single dose of AAV gene therapy and is comparable to chronic hydroxocobalamin administration
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Post-Doc Associate
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ASGCT 23rd Annual Meeting  webinar platform hosts Gene Therapy for Inborn Errors of Metabolism: New Approaches
The full session archive, available exclusively for attendees of the American Society of Gene & Cell Therapy's 23rd Annual Meeting, May 12-15, 2020.
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