Washington: Scientists have developed a gene editing therapy that can slow down ageing in mice with progeria syndrome, a rare genetic disorder that also afflicts humans. The research provides insights into the molecular pathways involved in accelerated ageing, as well as how to reduce toxic proteins via gene therapy.
Ageing is a leading risk factor for a number of debilitating conditions, including heart disease, cancer and Alzheimer’s disease. This makes the need for anti-ageing therapies all the more urgent.
Scientists from the Salk Institute in the US highlight a novel CRISPR/Cas9 genome-editing therapy that can suppress the accelerated ageing observed in mice with Hutchinson-Gilford progeria syndrome, a rare genetic disorder that also afflicts humans.
“Ageing is a complex process in which cells start to lose their functionality, so it is critical for us to find effective ways to study the molecular drivers of ageing,” said Juan Carlos Izpisua Belmonte, a professor at Salk’s Gene Expression Laboratory. Both mice and humans with progeria show many signs of ageing, including DNA damage, cardiac dysfunction and dramatically shortened life span.
The LMNA gene normally produces two similar proteins inside a cell: lamin A and lamin C. Progeria shifts the production of lamin A to progerin. Progerin is a shortened, toxic form of lamin A that accumulates with age and is exacerbated in those with progeria.
“Our goal was to diminish the toxicity from the mutation of the LMNA gene that leads to accumulation of progerin inside the cell,” said Hsin-Kai Liao, a staff researcher in the Izpisua Belmonte lab.