High cholesterol: treatment resulting from Crispr-Cas9

A recent study by scientists stated that they will use a new method of genetic editing, the so-called base editing. It is based on the same principles as Crispr-Cas9 which will not cut the double strand of DNA instead replacing only the damaged nucleotides. It will also be used to treat genetic hypercholesterolemia ( raised bad blood cholesterol ). In practice, there will be no more cutting and pasting of DNA.

Science will now take advantage of a new version of gene editing. A method similar to molecular scissors, but which will convert defective genetic elements into healthy molecules by treating genetic diseases. Remembering what Crispr-Cas9 is, it can be said that it is the revolutionary technique of editing DNA sequences for the correction of 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 stitching together filaments without errors.

However, this method is not perfect, and since 2016 we have been working on a new version of genetic editing, called base editing. It works in the opposite way to Crispr-Cas9: it does not modify the DNA with cut and paste. Instead, it couples a Cas9 protein to another enzyme capable of converting one letter of DNA into another. Cas9 guides the modifying enzyme to the appropriate location in the genome to allow the necessary modification to be made. This is why this technique seems to be tested to treat familial hypercholesterolemia.

The project envisages the injection of a substance that inhibits the central gene in hypercholesterolemia, that is, a regulator of cholesterol in the blood. Thus, the modification made to human DNA will be useful in permanently lowering the levels of bad cholesterol that clogs the arteries. If the effectiveness in animals will also occur in humans, then this method could also prevent strokes and heart attacks . In addition, becoming an aid for rare diseases and without side effects.



  • The new potential treatment for high cholesterol? It could come from a “cousin” of Crispr-Cas9 (ohga.it)

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