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Gene Therapy

UT SouthWestern, Boston Children's Hospital & NIH

Gene therapy is a ground breaking approach to treating monogenetic diseases. It leverages a one-time delivery of a normal copy of the defective/missing gene causing the body to generate the naturally missing protein.  Our hope is that by leveraging Gene Therapy we will be able to halt and or reverse the disease in children affected by SPG50. 

Gene Therapy
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