Early disease can be treated with gene therapy

Scientists have been able to reverse many signs of aging in cells from children with premature aging.

Scientists at Methodist Reseach Research Institute in Houston (USA) claim to have reversed many signs of aging in cells from children with premature aging (progeria).

Progeria is a rare disease caused by a mutation of the LMNA gene that causes aging and premature death in children with the disease . It is estimated that there are about 250 children in the world currently suffering from this disease.

According to Medical News Today, children with progeria at birth may seem normal, but within a year children will begin to have special physical signs such as slow growth rate, fast hair loss. , nose is narrow, face and jaw are small compared to the rest of the body.

Picture 1 of Early disease can be treated with gene therapy
The relationship between lifespan and telomere length in chromosomes of human cells.

Children with progeria will also develop serious heart and circulation problems, stiffness of their joints, and hip dislocations. Most patients die of heart attack or stroke at the age of 13-15 and only a very few live through adolescence.

In recent years, scientists have found an association between life expectancy and telomere length in chromosomes of human cells.

Telomeres are DNA fragments of DNA structure, located at both ends of the chromosome to protect the genome. Telomeres are often compared to plastic ends wrapped at the ends of shoelaces to protect them from fiber and bluntness.

Normally each cell division, a small piece of telomeres will be lost, causing telomeres to shorten with age. When these telomeres reach critical lengths, the cells will stop dividing and die in series, following a process called apoptosis .

Shorter telomere is associated with higher morbidity and shorter life expectancy. For this reason, telomere lengths considered as "biological clocks" can predict cell life time, as well as the life span of an organism.

It has been found in children with premature aging, the rate of wear at telomeres occurs much faster than usual.

The team used a new technology called RNA therapy , which takes small molecules into cells to change their gene expression.

In this way, they can help children's cells with premature aging to produce telomerase, an enzyme capable of lengthening telomeres.

Picture 2 of Early disease can be treated with gene therapy
Meghan Waldron (center) suffered from premature age participating in a children's reading program at Kee-wanee camp in the US on August 1 (Photo: TWITTER).

Within a few days of treatment, the cells showed significant changes affecting their lifespan and function.

Dr. John Cooke, the leader of the study, said they were surprised that only one treatment could be so effective.

"Our approach has a greater effect on all signs of cell aging. We have significantly improved the ability of cells to replicate and reverse the production of inflammatory proteins. Our therapy, the signs of cellular aging in research are reversed " - Dr. Cooke confidently said.

He suggests that because this study shows that RNA therapy can reverse the shortening of telomeres in the cells of premature children, it may also help "reverse many of the aging-related diseases." ".

He and his colleagues believe they can make these results more useful in the coming years.