Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome

Nat Med. 2019 Mar;25(3):423-426. doi: 10.1038/s41591-018-0338-6. Epub 2019 Feb 18.


CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Genetic Therapy / methods*
  • HEK293 Cells
  • Humans
  • Lamin Type A / genetics*
  • Lamin Type A / metabolism
  • Mice
  • Point Mutation
  • Progeria / genetics
  • Progeria / therapy*


  • LMNA protein, human
  • Lamin Type A
  • Lmna protein, mouse
  • prelamin A