CRISPR Technology

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a gene editing technology, which replicates the natural defense mechanism in bacteria to fight virus attacks, using a special protein called Cas9.

o It usually involves the introduction of a new gene, or suppression of an existing gene, through a process described as genetic engineering.

· CRISPR technology does not involve the introduction of any new gene from the outside.

o CRISPR-Cas9 technology is often described as ‘Genetic Scissors’.

· Its mechanism is often compared to the ‘cut-copy-paste’, or ‘find-replace’ functionalities in common computer programs.

· A bad stretch in the DNA sequence, which is the cause of disease or disorder, is located, cut, removed, and then replaced with a ‘correct’ sequence.

· The tools used to achieve this are biochemical i.e., specific protein and RNA molecules.

o The technology replicates a natural defense mechanism in some bacteria that uses a similar method to protect itself from virus attacks.

 Mechanism:

o The first task is to identify the particular sequence of genes that is the cause of the trouble.

o Once that is done, an RNA molecule is programmed to locate this sequence on the DNA strand, just like the ‘find’ or ‘search’ function on a computer.

o After this Cas9 is used to break the DNA strand at specific points, and remove the bad sequence.

o A DNA strand, when broken, has a natural tendency to re-attach and heal itself. But if the auto-repair mechanism is allowed to continue, the bad sequence can regrow.

· So, scientists intervene during the auto-repair process by supplying the correct sequence of genetic codes, which attaches to the broken DNA strand.

· It is like cutting out the damaged part of a long zipper and replacing it with a normally functioning part.

o The entire process is programmable, and has remarkable efficiency, though the chances of error are not entirely ruled out.

What is the Significance of CRISPR-based Therapeutic Solutions?

§ Specific Treatment: CRISPR aids in disease treatment by correcting the underlying genetic problem. CRISPR-based therapeutic solutions are not in the form of a pill or drug. Instead, some cells of every patient are extracted, the genes are edited in the laboratory, and the corrected genes are then re-injected into the patients.

o What is to be edited, and where, is different in different cases. Therefore, a specific solution needs to be devised for every disease or disorder that is to be corrected.

· The solutions could be specific to particular populations or racial groups since these are also dependent on genes.

· The changes in genetic sequences remain with the individual and are not passed on to the offspring.

§ Permanent Cure of Genetic Diseases/Anomalies: A vast number of diseases and disorders are genetic i.e.; they are caused by unwanted changes or mutations in genes.

o These include common blood disorders like sickle cell anemia, eye diseases including color blindness, several types of cancer, diabetes, HIV, and liver and heart diseases. Many of these are hereditary as well.

o CRISPR opens up the possibility of finding a permanent cure for many of these diseases.

o Deformities like stunted or slow growth, speech disorders, or inability to stand or walk arise out of abnormalities in gene sequences.

· CRISPR presents a potential treatment for the cure of such abnormalities as well.

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