Why rare genetic diseases are a logical focus for RNA therapies

Nature. 2019 Oct;574(7778):S16-S18. doi: 10.1038/d41586-019-03075-5.
No abstract available

Keywords: Drug discovery; Therapeutics.

MeSH terms

  • Genetic Diseases, Inborn / therapy*
  • Humans
  • Molecular Targeted Therapy / standards
  • Molecular Targeted Therapy / trends*
  • RNA / therapeutic use*
  • Rare Diseases / therapy*

Substances

  • RNA