Novel Gene Editing Therapy To Reverse Ageing By Salk Institute Scientists

Novel Gene Editing Therapy To Reverse Ageing By Salk Institute Scientists

Ageing is a risk factor for a variety of ailments, including cardiovascular disease, cancer, and Alzheimer’s disease. This increases the demand for therapies drastically. Scientists at the Salk Institute, United States have come with a novel CRISPR/Cas9 genome-editing therapy that may suppress accelerated ageing in mice with progeria syndrome – a genetic disorder which afflicts humans.

A study published in journal Nature Medicine highlights the molecular pathways involved in accelerated ageing and also focuses on ways on how to minimize the toxic proteins via gene therapy.

As per Juan Carlos Izpisua Belmonte – a professor at Salk’s Gene Expression Laboratory, Progeria is an ideal disease model to study ageing as it allows to devise an intervention, refine and to test it again in a shorter time period.

Progeria With fast progression is one of the most severe forms of degenerative disorders caused by a mutation in the LMNA gene. Signs of ageing can be seen in Both mice, as well as humans with progeria which includes DNA damage, cardiac dysfunction, and dramatically shortened life span.

Normally two similar proteins are produced by the LMNA gene inside a cell: lamin A and lamin C. Progeria shift

the production of lamin A to progerin. Progerin is a short toxic form of lamin A that accumulates with age and is exacerbated in those with progeria.

A staff researcher named Hsin-Kai Liao- from Izpisua Belmonte lab stated that – the main aim behind this study was to remove the toxicity from the mutation of the LMNA gene which is the prime cause for accumulation of progerin within the cell.

In order to deliver the gene therapy into the cells of the progeria mouse model CRISPR/Cas9 system was utilized for expressing Cas9.

It was observed After two months from the delivery of the therapy that the mice were stronger and more active with an improvement in cardiovascular health as well. The mice showed decreased degeneration of a major arterial blood vessel and an abnormally slow heart rate which are the two issues commonly observed in progeria and old age.

After the study, it was seen that the treated progeria mice had activity levels similar to normal mice, and their life span was increased roughly by 25 percent.

Pradeep Reddy- a postdoctoral fellow from the Izpisua Belmonte lab stated that- they will be able to increase life span further once we improve the efficiency of our viruses to infect a wide range of tissues.