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US scientists unveil powerful new tools to fix genetic faults

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US scientists unveiled two new molecular editing tools designed to fix mutations that cause the majority of human genetic diseases, some of which have no known treatment. One technique, by David Liu of Harvard University and the Broad Institute of MIT and Harvard, offers a highly precise way to fix single-letter mistakes in genes, which are stretches of deoxyribonucleic acid or DNA.


A second, by Broad Institute molecular biologist Feng Zhang, focuses on editing ribonucleic acid or RNA, which carries the genetic instructions to make proteins, without altering DNA. Both techniques build off of the game-changing CRISPR-Cas9 gene editing tool, a type of molecular scissors for trimming unwanted parts of the human genome to replace with new stretches of DNA. The genome consists of six billion DNA letters, or chemical bases.


In a paper published on last Wednesday in the journal Nature, Liu and colleagues build on his pioneering work called base editing. Unlike CRISPR, which causes breaks in DNA, base editing chemically corrects single-letter errors in DNA.


“CRISPR is like scissors, and base editors are like pencils,” Liu said in a statement.


Base editing tinkers with DNA’s four chemical bases, adenine (A), cytosine (C), guanine (G) and thymine (T). It takes two bases to form base pairs which make up rungs in the spiral DNA ladder, and they follow specific rules — C pairs with G, and T pairs with A. Single-letter mistakes, called point mutations, can give rise to genetic diseases. Last year, Liu’s team described a base editor that could change CG base pairs into AT pairs.


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