Emerging Strategies for Genome Editing in the Brain

Trends Mol Med. 2018 Oct;24(10):822-824. doi: 10.1016/j.molmed.2018.07.008. Epub 2018 Aug 10.

Abstract

Despite the unparalleled therapeutic promise of genome editing, its curative power is currently limited by the substantial difficulty in delivering DNA-cutting enzymes to the cells in need of correction. A recent study demonstrates the potential for the delivery of pre-assembled genome-editing enzymes in the form of ribonucleoprotein complexes, which were used to rescue a mouse model of fragile X syndrome (FXS).

MeSH terms

  • Animals
  • Biological Transport
  • Brain / metabolism
  • Brain / pathology
  • CRISPR-Associated Protein 9 / genetics*
  • CRISPR-Associated Protein 9 / metabolism
  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Dependovirus / genetics
  • Dependovirus / metabolism
  • Disease Models, Animal
  • Fragile X Mental Retardation Protein / genetics
  • Fragile X Mental Retardation Protein / metabolism
  • Fragile X Syndrome / genetics
  • Fragile X Syndrome / metabolism
  • Fragile X Syndrome / pathology
  • Fragile X Syndrome / therapy*
  • Gene Deletion
  • Gene Editing / methods*
  • Gene Transfer Techniques*
  • Gold / administration & dosage
  • Gold / chemistry
  • Gold / metabolism
  • Humans
  • Metal Nanoparticles / administration & dosage
  • Metal Nanoparticles / chemistry
  • Mice
  • Mice, Knockout
  • RNA, Guide / genetics
  • RNA, Guide / metabolism
  • Ribonucleoproteins / genetics*
  • Ribonucleoproteins / metabolism

Substances

  • Fmr1 protein, mouse
  • RNA, Guide
  • Ribonucleoproteins
  • Fragile X Mental Retardation Protein
  • Gold
  • CRISPR-Associated Protein 9