Single-dose CRISPR-Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome

Nat Med. 2019 Mar;25(3):419-422. doi: 10.1038/s41591-019-0343-4. Epub 2019 Feb 18.


Hutchinson-Gilford progeria syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we show that reduction of lamin A/progerin by a single-dose systemic administration of adeno-associated virus-delivered CRISPR-Cas9 components suppresses HGPS in a mouse model.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Disease Models, Animal
  • Genetic Therapy / methods*
  • Lamin Type A / genetics*
  • Lamin Type A / metabolism
  • Longevity*
  • Mice
  • Mutation
  • Progeria / genetics*
  • Protein Isoforms / genetics
  • Protein Isoforms / metabolism


  • Lamin Type A
  • Lmna protein, mouse
  • Protein Isoforms