What is the role of gene therapy in the treatment of ataxia-telangiectasia?

Updated: Jul 28, 2020
  • Author: Andrew A Dahl, MD, FACS; Chief Editor: Hampton Roy, Sr, MD  more...
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Answer

The gene responsible for ataxia-telangiectasia, the ATM gene, was discovered in 1995. This gene makes a protein that activates a number of other proteins that control cell cycle, DNA repair, and cell death. There is ongoing preclinical and early clinical research on gene therapy to treat ataxia-telangiectasia and other diseases. [7]


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