Gene editing technologies such as CRISPR / Cas9 and its innovations have great potential in genetic screening, genetic diagnosis, especially the development of new therapies.

Emmanuelle Charpentier (left) and Jennifer Doudna take souvenir photos while attending an event at a park in Oviedo, Spain on October 21, 2015 - Photo: Reuters

On the afternoon of October 7 (Vietnam time), the Royal Swedish Academy of Sciences announced the 2020 Nobel Prize in Chemistry for two female scientists Emmanuelle Charpentier and Jennifer A. Doudna discovered one of the sharpest tools. of gene editing technology, which is CRISPR / Cas9.

"Now, with CRISPR / Cas9, scientists can change the 'code' of life (DNA) in just a few weeks" - said Göran K. Hansson, general secretary of the Royal Swiss Academy of Sciences Swedish, said.

“This technology has pioneered the sciences of life research, contributed to new cancer treatments and could turn the dream of curing genetic diseases into reality.

Committee announced the Nobel Prize said.

Genetic "scissors"

Using them, researchers can alter the DNA of animals, plants and microorganisms with extreme precision. These genetic "scissors" have brought life sciences into a new era and in many ways are bringing the greatest benefit to humanity.

Ever since Charpentier and Doudna discovered the CRISPR / Cas9 genetic scissors in 2012, their use has exploded. This tool has contributed to many important discoveries in basic research, and plant researchers have been able to develop crops that are resistant to fungi, pests and drought. This technology has had a revolutionary impact on life science and especially medicine.

Due to its many advantages in genomic editing, the CRISPR / Cas9 genetic pull has attracted considerable attention and scientists gradually see it as a powerful therapeutic tool for the treatment of related diseases. to genetic mutations. Thereby contributing to the development of therapeutic therapies, especially cancer and can help realize the dream of curing other genetic diseases.

Diseases using CRISPR / Cas9 are being tested in clinical trials such as cancer, sickle cell anemia, ß-thalassemia disease and even HIV infection.

CRISPR / Cas9 is a system that is likened to the immune system of bacteria to fight infection of foreign DNA molecules from viruses or other plasmids. Scientists apply CRISPR / Cas9 to design the desired genomic DNA editing system.

Helps cure many diseases, including cancer

In cancer, mutations of oncogene and tumor suppressor genes offer great opportunities for using genome-modifying methods. The ultimate goal of CRISPR / Cas9 cancer therapy is to remove malignant mutations and replace them with normal DNA sequences.

The recent application of CRISPR / Cas9 technology to create CAR-T cells to attack malignant cells offers great hope in cancer therapies. Since the beginning of 2020, more than 11 clinical trials have been conducted to evaluate the effectiveness of the CRISPR / Cas9 system in cancer treatment.

Heart-related diseaes. Different types of cardiovascular disease often involve a single inherited mutation or a combination of rare inherited heterozygous mutations. In fact, clinical treatments focus on relieving disease symptoms without addressing potential genetic defects.

Currently, gene editing technology helps to establish disease patterns as well as in-depth analysis of pathogenic genes and their molecular mechanisms, thereby helping to develop gene therapies in controlling gene expression. can and improve gene function.

Neurodegenerative diseases, such as Huntington's disease, Alzheimer's disease, and Parkinson's disease, are a group of conditions that attract the most attention because no specific diagnostic or treatment has been established.

Many studies show that neurodegenerative diseases are caused by the complex interactions of many genetic factors. The emergence of gene editing platforms offers a convenient approach to the study of gene function and therapy related to these diseases.

Diseases caused by viruses. Gene editing platforms have emerged recently as an antiviral therapy for the treatment of infectious diseases, by altering the host genes that viruses attack or by targeting the genes of essential viruses directly. for their reproduction.

To date, genomic-based HIV therapy involves generating HIV-resistant CD4 + T-cells that modify the genes involved in infection and then reuse the cells. editing into patient. Laboratory results show that CRISPR / Cas9 can not only specifically eliminate latent HIV infection, but can also prevent new HIV infection.

In addition, the laboratory studies also showed that the use of the CRISPR / Cas9 gene editing tool is capable of identifying and cleaving the genome of the HPV virus (causing cervical cancer) or HBV (causing hepatitis B).

To summarize, gene editing technology such as CRISPR / Cas9 and its innovations have great potential in promoting development in life science, especially genetically engineered, exogenous applications, as well as functional gene screening and genetic diagnosis, particularly the development of new therapies.

Emmanuelle Charpentier, 52 years old, is a French doctor and research specialist, working in microbiology, genetics and biochemistry. Since 2015, she has been the director of the Max Planck Institute for Infectious Biology in Berlin, Germany. In 2018, she established an independent research institute - Max Planck Unit specializing in pathogen research.

Jennifer Anne Doudna, 56, is an American biochemistry researcher known for her leading role in gene editing technology (CRISPR). She is a leading professor at the Li Ka Shing Biological and Medical Science Center of the University of California, Berkeley, USA.

Source: //tuoitre.vn/cong-nghe-chinh-sua-gen-gianh-nobel-hoa-hoc-2020-giup-chua-nhieu-benh-gom-ca-ung-thu-20201008090046505.htm