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CRISPR: The Future of Gene Therapy

The world of medicine and science is ever-advancing and constantly surprises modern society with its newest inventions. In the early 1990s, an especially unique and life-changing technology was engineered and formed the basis for a change in human life.

CRISPR, a term used in microbiology, describes an adaptive immune system. CRISPR stands for Clustered Regularly-Interspaced Short Palindromic Repeats and categorizes natural segments of genetic code found in prokaryotes. These reiterations are copied in small pieces of bacteria’s DNA to help them develop immunity to viruses. The small repeated sections of DNA allow organisms to remember invaders and ward them off in subsequent encounters, thus playing a pivotal role in antiviral defense mechanisms. Now, this same system has been developed into a genome editing tool and represents the work of many scientists.

In recent scientific development, CRISPR has been used in the generation of CRISPR-Cas, a tool used for the modification of genes. Cas9 is a vital RNA-associated protein also used as a defense mechanism against foreign invaders. This enzyme has the ability to cut DNA and, therefore, destroys viruses that pose harm to bacteria. The Cas9 nuclease has two active cutting sites to complement each strand of the double helix structure. With the help of a guide RNA, which contains the previously copied and stored gene sequence of the invading pathogen, Cas9 unwinds foreign DNA and verifies whether or not it is complementary to the spacer region RNA. If this DNA sequence matches that of the known virus, Cas9 chops up the intruder, thus protecting the body against future infection.

While the idea of gene therapy has been prevalent for several years, CRISPR-Cas was first described in 2012 and 2013, when scientists realized that the combination of the Cas9 enzyme and the guide RNA had the ability to dramatically alter the genomes of organisms. This device can effectively tweak most genetic code in almost any plant or animal, thereby significantly reconstructing inherited traits. The cuts made by this tool can turn various genes on and off or can repair genetic mutations. CRISPR-Cas holds tremendous power over humanity as science, now, has the ability to change the genetic makeup of a vast array of organisms. On one end of the spectrum, this tool can be used to cure genetic diseases, such as Sickle Cell Anemia. In a more extreme sense, this technology can be used to engineer ideal characteristics in humans. These advances, however, are seemingly far off and pose no current threat to society. Now, CRISPR-Cas is mostly used in basic biology laboratories for conducting research but has been used in some cases to prepare animal organs for human transplant surgeries. CRISPR-Cas is a groundbreaking technology that could revolutionize the scientific world and humanity as a whole.

Picture Source: Animated biology With arpan on youtube