In its five years of existence, CRISPR/Cas9 has revolutionized the field of gene editing, allowing researchers to edit DNA like a piece of text. While it’s potential is unquestionable, ethics and safety concerns have prevented CRISPR from being used to treat human diseases. Now, scientists at the Salk Institute have found a way around one of those hurdles, modifying the CRISPR system to treat several diseases in mice without cutting DNA, which means they avoid unwanted mutations. The technology works epigenetically, influencing gene activity without changing the DNA sequence.
To transport this bulkier CRISPR system into cells, the researchers delivered its components separately, attaching them to two viruses. They engineered the adapted system to activate specific genes that could reverse the symptoms of each targeted disease. In a mouse model of type 1 diabetes, for example, the team activated genes that could generate insulin-producing cells. The treatment worked, lowering blood …
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