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Tue Oct 22, 2019, 04:52 AM

Prime editing: DNA tool could correct 89% of genetic defects

https://www.bbc.com/news/health-50125843

Prime editing: DNA tool could correct 89% of genetic defects

By James Gallagher
Health and science correspondent

21 October 2019

A new way of editing the code of life could correct 89% of the errors in DNA that cause disease, say US scientists. The technology, called prime editing, has been described as a "genetic word processor" able to accurately re-write the genetic code.

It has been used to correct damaging mutations in the lab, including those that cause sickle cell anaemia. The team at the Broad Institute say it is "very versatile and precise", but stress the research is only starting.
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The study, in the journal Nature, used prime editing to accurately insert or delete sections of DNA; as well as correct typos in a single "letter" out of the three billion that make up the human genetic code.
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The technology uses a lab-made sequence of genetic code. This has two roles, one is to find the specific part of the DNA you want to edit and the other contains the change you want to make.

This is paired with an enzyme (called a reverse transcriptase) which copies the relevant edits over into the DNA.
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The challenge - as with all other gene editing technologies - will be getting the molecular machinery that is capable of performing these edits into the right parts of the human body and to ensure they are safe.

Like other gene-editing technologies, it is likely the first applications will be in diseases where cells can be taken out of the body, edited, checked to ensure they are safe and put back in. That would apply to some blood disorders like sickle cell or thalassemias, where bone marrow can be removed and put back in.
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