What is the role of gene therapy in the treatment of osteogenesis imperfecta (OI)?

Updated: Feb 24, 2020
  • Author: Manoj Ramachandran, MBBS, MRCS, FRCS; Chief Editor: Harris Gellman, MD  more...
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Answer

Gene therapy is being explored in animal models, but major obstacles remain, both because of intrinsic difficulties (as illustrated by attempts to treat conditions such as cystic fibrosis) and because of the dominant negative mechanism of the disease. The success achieved in treating X-linked severe combined immunodeficiency disease (X-SCID) by means of gene therapy provides some hope that this approach may eventually be successful in conditions such as OI.


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