How Gene Editing Technology Is Helping In Disease Prevention?

Gene editing technology has opened new doors in the field of disease prevention. First implemented in 2012, CRISPR gene-editing has exploded in popularity owing to its capacity of making the procedure faster, cheaper and easier. Today, a range of complications starting with cancer to blindness and cardiovascular disease can be cured by implementing gene editing technology. The novel CRISPR gene-editing technology has brought on the table a new potential to edit and genetic mutation at will to cure a range of gene related diseases. Here few examples of gene-editing technologies that are giving positive results in different disease prevention.

Potential in Cardiovascular Disease The main causes of death globally, according to 2015 data from the World Health Organization, are ischemic heart disease and stroke. In that year, 17.7 million individuals died from cardiovascular disease, with at least 75 percent of those fatalities taking place in low- and middle-income nations. Although antibody-based therapies have been launched to help those most at risk, the cost and complexity of the treatments means that a simpler, one-off fix such as a vaccine would be of benefit to many more people around the world.

The good news is that gene discovery and the development of genome-editing tools like CRISPR-Cas9 gene-editing technology have increased the likelihood that this future vision of a vaccine-led approach to preventing heart disease will materialize. When scientists looked at three French families with people who had potentially fatal amounts of low-density lipoprotein (LDL) cholesterol and who also had a mutation in the PCSK91 gene, they made their first significant discovery. An enzyme that controls the amount of LDL, or "bad" cholesterol, is encoded by PCSK9. The families' mutations boosted the enzyme's activity, which raised the blood's amount of LDL cholesterol. Therefore, reducing PCSK9 such that the enzyme it encodes no longer functions might lower LDL cholesterol levels.

Enhancing the Cure for Metabolic Disease Latest genome editing technologies, such the CRISPR/Cas9 platform, allow for the precise and long-lasting change of the genome in a cell or living thing. With this method, DNA may be added, removed, or replaced at a particular location in the genome. As a result, during cell duplication, the altered DNA is permanently passed on to daughter cells. New therapeutic options for treating and curing human illnesses are made possible by this site-specific repair of disease-causing mutations. Although the method is effective in animal models, it needs more work before it can be widely applied in the clinic. This work will help to control the method's expression of the therapeutic endonuclease, limit its undesirable off-target activity, and reduce the amount of virus that is used.

A diverse group of inherited human illnesses known as inborn errors of liver metabolism are defined by damaged proteins that are involved in the synthesis or catabolism of amino acids, lipids, or carbohydrates. In addition, they cause a variety of metabolic issues that cause people to complain in varying degrees. About 10% of pediatric liver transplants are caused by inborn metabolic abnormalities, which account for a considerable share of juvenile diseases globally. Inborn errors of liver metabolism are an appealing class of disorders that may be addressed by gene editing and treatment technologies since the condition is brought on by the alteration of a single gene.

Advancing Cancer Research The direction of cancer research has changed as a result of the realization that DNA alterations are the root of cancer. Thanks to CRISPR technology, researchers can now edit the DNA of human cells with the precision and ease of a pair of scissors, advancing cancer research to a new level. The CRISPR tool in the lab is made up of two essential components: a guide RNA and a DNA-cutting enzyme, most usually Cas9. Scientists developed the guide RNA to resemble the DNA of the target gene. As implied by its name, the guide RNA works with Cas to point Cas in the direction of the target. When the guide RNA and DNA align, Cas begins to cut the target gene's DNA.

“CRISPR gene-editing technology is becoming a mainstream methodology used in many cancer biology studies because of the convenience of the technique,” said Jerry Li, M.D., Ph.D., of NCI’s Division of Cancer Biology. Depending on the CRISPR tool being used, the following step varies. In a few rare circumstances, the target gene's DNA is damaged during repair, making the gene inactive. Other CRISPR variants allow for more specific gene editing, such as the addition of a new DNA segment or the modification of a single DNA letter. CRISPR is now being tested on cancer patients instead than just in test tubes. For instance, in a tiny trial, scientists examined an immunotherapy for cancer that used immune cells that had undergone CRISPR editing to enhance their ability to detect and combat cancer.

In the Future Although the concept of gene-editing is comparatively new in the field of medical science it is already providing positive results in multiple domains. Starting with cancer treatment to cardiovascular issues, gene-editing will provide a major leap forward to humanity as in all. In the coming days, gene editing will provide a way to target and destroy complex diseases such as cancer and AIDS, and even target genes associated with mental illnesses.