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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Infographic On MSUD 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.