This is a revolution in medicine. Presumably, a third of all DNA errors can be edited using this technology.
A group of employees of Harvard University announced the creation a "basic editor" of DNA that can cut and replace defective genes.
This is an amazing discovery that could turn the whole of medicine upside down.
Last year, they conducted experiments on adult mice with progeria, a rare genetic disease that causes premature aging of the body. It is caused by the mutation of a single gene, and has devastating consequences.
After injections of drugs, 90 percent of the rodents recovered muscle cells, their life expectancy doubled.
Here is how geneticist David Liu, the leader of this study, explains everything: DNA has 4 nitrogenous bases of nucleotides: adenine (A), cytosine (C), guanine (G) and thymine (T). They form specific pairs that must always coincide with each other: A with T and G with C.
But often there are "spelling errors" that cause genetic diseases. The gene editor "goes into the animal's cells, looks for the error (in progeria it's a C instead of a T), and changes the C back to a T."
Liu's team also found that basic editors also work if you "cut" unedited strand of the DNA double helix, causing the cell to copy the edited portion to the second thread.
"And it's all. We never go back to the patient a one-time treatment that permanently eliminates the disease-causing mutation', Liu said.
The technology was then tested on mice with sickle cell anemia. This is a fairly common hereditary blood disease. 16 weeks after the start of the experiments, 80 percent of the animals were already healthy.
More than 75 thousand such "spelling" errors are now known. And about a third of them can be corrected using such an editor.
What Diseases Can Gene Editing Help Defeat?
Genetic editing cannot cure all diseases. For example, infections and cancer cells are two areas that are not well suited for gene editing because you would need to touch each cell to stop the disease.
"But in many genetic diseases, we often only need to edit 20% or 30% of the tissue," says Dr. Liu.
Scientists hope that technology will help with Alzheimer's disease and other types of dementiawhich now, with a rapidly aging population, are becoming increasingly relevant.
We all have a predisposition to certain diseases. If such an editor is launched into our cells, then bad genes will be neutralized, and the person will become healthy.
For example, migraine due to the presence of genes that produce proteins that act on certain receptors and lead to the development of pain. Editing these genes will stop protein synthesis and the pain will disappear.
Your Doctor Pavlova
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