High Cholesterol: Treatment Due to Crispr-Cas9

A recent study by scientists states that they are going to use a new method of genetic editing, called base editing. It is based on the same principles as: Crispr-Cas9 which will not cut the DNA double strand, but will only replace the damaged nucleotides. In addition, it will be used for the treatment of thegenetic hypercholesterolemia (increase cholesterol bad in the blood). In practice, there will be no more cutting and pasting with DNA.

Science will now benefit from a new version of gene editing. A method similar to molecular scissors, but converting defective genetic elements into healthy molecules by treating genetic diseases. If we remember what Crispr-Cas9 is, it can be said that it is the revolutionary technique of creating sequences of DNA for correcting genetic errors. With this technique, both DNA strands can be broken at the modification point. This allows the cell’s DNA to be repaired by bonding filaments together flawlessly.

However, this method is not perfect and since 2016 we have been working on a new version of genetic editing called base editing. It is counterproductive to Crispr-Cas9: it does not modify DNA by cutting and pasting. Instead, it pairs a Cas9 protein to another enzyme capable of converting one letter of DNA into another. Cas9 directs the modifying enzyme to the correct location in the genome to allow for the necessary modification. This is why this technique appears to have been tested to treat familial hypercholesterolemia.

The project aims to inject a substance that inhibits the central gene in hypercholesterolemia, ie a regulator of cholesterol in the blood. Thus, the modification made to human DNA will be helpful in permanently lowering the levels of bad cholesterol that clog the arteries. If the effectiveness in animals also occurs in humans, then this method can also: prevent strokes and heart attacks. In addition, become an aid in rare diseases and without side effects.