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Prime Editing Of DNA Might Offer Cure For Diseases In Humans

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Scientists in the U.S. have developed a new gene-editing system that could be more precise and efficient than current technology like CRISPR-Cas9, thus substantially expanding the scope and capabilities of genome editing.

The new technology, called prime editing, is very versatile and precise, according to the scientists. It can make extremely precise edits of the code of life, or DNA.

Prime editing was developed by a team of researchers at the Broad Institute of MIT and Harvard University.

Scientists expect that, in principle, prime editing can correct about 89 percent of the about 75,000 known pathogenic human genetic mutations that can cause disease in people. However, they added that the research is only starting.

The research was published in the journal 'Nature' on October 21.

The fundamental building blocks of DNA are the four bases - Adenine, Cytosine, Guanine and Thymine. They are commonly designated by the letters, A, C, G and T. Three billion of these letters make up the human genetic code.

Genome editing, or gene editing, is a type of genetic engineering in which DNA bases are inserted, deleted, modified, or replaced in the genome of a living organism.

Current gene-editing technologies like CRISPR-Cas9 and base editors are powerful, but they are seen as lacking precision and they also suffer from high error rate.

According to the research, prime editing can be used to accurately insert or delete sections of DNA.

The scientists said they performed more than 175 DNA edits in human cells. They were able to correct the genetic mutations for two diseases - sickle cell and Tay-Sachs disease.

"Prime editing offers efficiency and product purity advantages over homology-directed repair, complementary strengths and weaknesses compared to base editing, and much lower off-target editing than Cas9 nuclease at known Cas9 off-target sites," the scientists said.

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