David Liu innovations in the field of DNA draws the future of medicine

On Saturday, a prize is awarded as “Oscar of Science” to the American molecular biology scientist David Liu, for his basic contribution to the great progress in the field of genetic therapy, as his research allows for unprecedented DNA, which opens the door to a radical transformation of medicine.

A professor at the Massachusetts Institute of Technology (MIT) and Harvard University in the northeastern United States won the “Brothers” Scientific Prize for creating two techniques, one of which has already improved the lives of patients with serious genetic diseases, and the other is a major development in the field of genome liberation.

“The conversion of a sequence of DNA into a new sequence of our choice is a great ability,” the 51 -year -old scientist told AFP before the eleventh ceremony for the distribution of these awards in Los Angeles.

Leo plans to donate his charitable association with the largest department of a prize worth three million dollars to obtain from this prestigious event, which was launched by entrepreneurs in Silicon Valley at the beginning of the second decade of the twenty -first century to reward the achievements in the field of basic research.

The researcher was given this award for his research in the field of “basic editing” and “primary editing” of the genome, which are techniques with several possible applications in medicine as well as in agriculture, for example to develop crops with a higher food value or with greater ability to endure.

Molecular scissors

DNA consists of four types of nucleotides that make up the letters of the genetic alphabet are “A”, “C”, “T” and “G”. The inscriptions in their compositions and sequences are the cause of thousands of genetic diseases, which were only a limited number of them to this day, through the release of the genome.

The revolutionary scissors technology “Krisper-Cas 9”, which won the Nobel Prize in Chemistry in 2020, has sparked great hopes in this field. However, this technique has its limits, as it cuts the two series that form the double spiral structure of the DNA, which contributes to disrupting genes instead of correcting it, with the risk of introducing new errors.

However, the treatment of genetic diseases requires “in most cases, ways to correct errors in the DNA sequence, not just disrupting the gene with imbalance.”

This observation prompted Liu to work on “basic liberation”. This technology uses a modified version of “Cass 9” within the framework of molecular scissors technology. This time, the protein no longer cuts the two DNA series, but still targets a specific genome serial, and it is integrated with an enzyme capable of converting nelotide or one letter to another.

While most traditional genetic treatments aim to disrupt pathogenic genes, “basic liberation” can directly fit about a third of the relevant mutations.

This innovative technique is at least 14 clinical trials, one of which allowed the treatment of patients suffering from alpha-1 antitribebsin, a rare genetic disease that affects the liver and lungs.

“Small miracles”

Although the “basic liberation” technique is promising, it will not be able to treat only about 30% of about 100,000 mutamors that cause genetic diseases.

It is not useful, for example, if there is a missing or additional letter within the DNA sequence.

In order to address these defects, the David Leo team announced in 2019 the “initial editing” technology, a method capable of replacing the entire DNA sequences.

While the “Krisper-Cas 9” technique can be likened to DNA scissors, and “basic editing” with an eraser that allows one letter to correct each time, the “initial editing” is closer to the function of “research and replacement” in the text processor.

The creation of this revolutionary tool is possible thanks to a series of discoveries that the Leo team described as “small miracles”. The scientist emphasized that this technique is so far “the means with the most intense capabilities to modify the human genome.”

It can be used in particular for the treatment of cystic fibrosis, a genetic disease that leads to the deterioration of the respiratory system and the digestive system, and it often occurs due to the deletion of three nucleotides.