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.