4.5 Review

Genetic therapy for congenital myopathies

Journal

CURRENT OPINION IN NEUROLOGY
Volume 34, Issue 5, Pages 727-737

Publisher

LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/WCO.0000000000000978

Keywords

antisense oligonucleotides; congenital myopathies; clustered regularly interspaced short palindromic repeats gene editing; Gene therapy

Funding

  1. National Institutes of Health (NIH)
  2. Canadian Institutes of Health Research (CIHR)
  3. Muscular Dystrophy Canada
  4. Foundation Building Strength
  5. Myotubular Trust

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The field of genetic therapies is rapidly advancing, with the first gene-based treatments now in clinical practice. While there are currently no approved drug therapies for congenital myopathies, several candidate therapeutics, including gene replacement therapy and antisense oligonucleotide-based gene knockdown, are in development. Genetic therapies are bringing precision medicine to neurological diseases, with congenital myopathies being ideally suited for these approaches.
Purpose of review There has been an explosion of advancement in the field of genetic therapies. The first gene-based treatments are now in clinical practice, with several additional therapeutic programs in various stages of development. Novel technologies are being developed that will further advance the breadth and success of genetic medicine. Congenital myopathies are an important group of neuromuscular disorders defined by structural changes in the muscle and characterized by severe clinical symptoms caused by muscle weakness. At present, there are no approved drug therapies for any subtype of congenital myopathy. In this review, we present an overview of genetic therapies and discuss their application to congenital myopathies. Recent findings Several candidate therapeutics for congenital myopathies are in the development pipeline, including ones in clinical trial. These include genetic medicines such as gene replacement therapy and antisense oligonucleotide-based gene knockdown. We highlight the programs related to genetic medicine, and also discuss congenital myopathy subtypes where genetic therapy could be applied. Genetic therapies are ushering in an era of precision medicine for neurological diseases. Congenital myopathies are conditions ideally suited for genetic medicine approaches, and the first such therapies will hopefully soon be reaching congenital myopathy patients.

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