What is the role of genetic therapy in the treatment of spinal muscle atrophy (SMA)?

Updated: Nov 05, 2018
  • Author: Ashish S Ranade, MBBS, MS, MRCS; Chief Editor: Jeffrey A Goldstein, MD  more...
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Answer

Research into genetic therapies, as well as molecular and stem cell–mediated therapies, is ongoing. [34, 35, 36]  The Cure SMA drug pipeline has identified four possible treatment targets [37] :

  • Replacement or correction of the faulty  SMN1 gene
  • Modulation of the low-functioning  SMN2 “backup gene”
  • Neuroprotection of the motor neurons affected by loss of survival motor neuron (SMN) protein
  • Muscle protection to prevent or restore the loss of muscle function in SMA

Proper care can improve quality of life for those with SMA.


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