Understanding Gene Therapy


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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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.