For the April edition of the Journal Club, we discussed the recent paper from the Salk Institute. The findings, published in the journal Nature Medicine, showcase a novel CRISPR/Cas9 genome-editing therapy that can suppress the accelerated aging observed in mice with Hutchinson-Gilford progeria syndrome. This treatment provides an important insight into the molecular pathways involved in accelerated aging, as well as how to reduce toxic proteins via gene therapy. The researchers hope to translate this therapy to humans to potentially provide a cure for progeria as well as possibly slowing down the aging process to delay the onset of age-related diseases in everyone.
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.
Beyret, E., Liao, H. K., Yamamoto, M., Hernandez-Benitez, R., Fu, Y., Erikson, G., … & Belmonte, J. C. I. (2019). Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome. Nature medicine, 1.