Understanding Gene Therapy


Experimental, molecular therapies, including delivery of messenger RNA (mRNA) and AAV gene therapy strive to overcome the genetic mutation(s) causing MSUD in different ways. To download this infographic, click here.





What Is Gene Therapy? How Does It Work?

U. S. Food and Drug Administration


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Genetic Therapies

National Institutes of Health 


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Gene & Cell Therapy Education


American Society of Gene + Cell Therapy


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