In 2023, the Nobel Prize in Physiology or Medicine awarded the Nobel Prize in Physiology or Medicine to the inventor of RNA interference technology, Vasse Glubos, and Lotschickel of the Sinak Institute of Molecular Technology.
This incident also means that nucleic acids are gradually changing from "niche" to "popular", and people are very curious about nucleic acids.
What is nucleic acid, and why can nucleic acid become the darling of the medical field?
1. What is nucleic acid?
Nucleic acids are long chains of inosinate groups that are important components in living organisms, including DNA and RNA. DNA is the material that carries genetic information in living organisms, while RNA is mainly responsible for copying DNA information, transmitting DNA information, and converting it into proteins. It can be seen that DNA is the "big housekeeper" and RNA is the "little housekeeper". So what is a nucleic acid drug?
Researchers have found that RNA can be used not only to transmit genetic information, but also to precisely regulate specific proteins. The researchers translated this discovery into a new drug technology that uses the properties of RNA to act directly on the target molecule to develop a unique, customized gene therapy technology.
Nucleic acid drugs are a major innovation in the field of gene therapy and precision targeted therapy. Different from traditional small molecules, antibodies, vaccines, and protein drugs, nucleic acid drugs can directly interfere with the functions of DNA and RNA, with stronger specificity and deeper regulatory levels.
Why are nucleic acid drugs gaining momentum?
A nucleic acid drug is a drug that can target specific genes, which has higher precision and specificity than traditional drugs. Because nucleic acid drugs can interfere with and regulate specific genes, they can not only be used to treat abnormal genes in the human body, but also to inhibit the abnormal expression of genes.
With the continuous deepening of basic research, people have a deeper understanding of the relationship between genes and diseases. Many diseases are caused by genetic mutations, and the purpose of treating the disease can be achieved by repairing or suppressing the abnormal gene. Therefore, nucleic acid drugs can be used to treat various rare diseases, genetic diseases and some common diseases.
On the other hand, nucleic acid drugs can also be used to suppress the development of diseases. The normal physiological activity of cells depends on correct gene expression, and abnormal gene expression may lead to abnormal state of cells, which can eventually lead to the occurrence of diseases. By inhibiting abnormal gene expression, the purpose of treating diseases can be achieved.
At present, a series of major breakthroughs have been made in the research and development of nucleic acid drugs, and many new nucleic acid drugs have entered clinical trials and are expected to be launched in the future. It is expected that in the future, nucleic acid drugs will inject new impetus into the development of gene therapy and precision medicine, and provide more and better options for the treatment of various diseases.
Why are nucleic acid drugs attracting attention?
As a new type of drug, nucleic acid drugs have many advantages that traditional drugs do not have. First, nucleic acid drugs have higher specificity. By precisely targeting specific genes or RNA molecules, nucleic acid drugs can achieve "magic knife" gene therapy, and nucleic acid drugs are more specific to the molecules that directly act, and have little impact on normal molecules.
Second, nucleic acid drugs have higher precision. Traditional drugs achieve efficacy by interfering with proteins outside the cell, but nucleic acid drugs act directly on nucleic acid molecules inside the cell, which can more precisely regulate biological processes at the molecular level.
Finally, nucleic acid drugs have higher plasticity. While traditional drugs are difficult to change the molecular structure once the molecular structure is determined through chemical synthesis, nucleic acid drugs can be designed according to the sequences of different genes, and their structure and function can be customized as needed.
Due to these advantages, nucleic acid drugs have attracted much attention in the fields of basic research and clinical treatment. Many researchers believe that nucleic acid drugs are expected to become an important means of treating cancer, genetic diseases, autoimmune diseases and other diseases in the future. It is believed that with the continuous advancement of technology, nucleic acid drugs will play an increasingly important role in the future.