Failure of genetic therapies for Huntington's devastates community

Nature. 2021 May;593(7858):180. doi: 10.1038/d41586-021-01177-7.
No abstract available

Keywords: Drug discovery; Gene therapy; Neurodegeneration.

Publication types

  • News

MeSH terms

  • Disease Progression
  • Female
  • Genetic Therapy / adverse effects*
  • Hope
  • Humans
  • Huntingtin Protein / deficiency
  • Huntingtin Protein / genetics
  • Huntingtin Protein / metabolism
  • Huntington Disease / genetics*
  • Huntington Disease / therapy*
  • Mutant Proteins / genetics
  • Mutant Proteins / metabolism
  • Oligonucleotides / administration & dosage
  • Oligonucleotides / adverse effects*
  • Oligonucleotides / therapeutic use*
  • Oligonucleotides, Antisense / administration & dosage
  • Oligonucleotides, Antisense / adverse effects*
  • Oligonucleotides, Antisense / therapeutic use*
  • Optimism
  • Treatment Failure

Substances

  • HTT protein, human
  • Huntingtin Protein
  • Mutant Proteins
  • Oligonucleotides
  • Oligonucleotides, Antisense
  • tominersen