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Postnatal gene therapy for neuromuscular diseases - opportunities and limitations.

Janbernd Kirschner
Published in: Journal of perinatal medicine (2021)
During the last decade a number of innovative treatments including gene therapies have been approved for the treatment of monogenic inherited diseases. For some neuromuscular diseases these approaches have dramatically changed the course of the disease. For others relevant challenges still remain and require disease specific approaches to overcome difficulties related to the immune response and the efficient transduction of target cells. This review provides an overview of the current development status of mutation specific treatments for neuromuscular diseases and concludes with on outlook on future developments and perspectives.
Keyphrases
  • gene therapy
  • immune response
  • induced apoptosis
  • cell cycle arrest
  • signaling pathway
  • oxidative stress
  • toll like receptor
  • cell proliferation
  • dna methylation
  • cell death
  • replacement therapy