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Gene Therapy
Gene Therapy (TSHA-102) for Rett Syndrome
Recruiting1 awardPhase 1 & 2
St. Paul, Minnesota
This trial is testing a new gene therapy called TSHA-102 in adult women with Rett syndrome. The therapy aims to fix the genetic problems causing the disorder by adding healthy genes to their cells. Researchers will study its safety and effectiveness over several years. TSHA-102 is a gene therapy aimed at addressing the genetic deficiencies in Rett syndrome by adding healthy MECP2 genes to the cells.
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