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Two studies published yesterday (October 9) in Nature Medicine report success using modified CRISPR-Cas9 gene editing to prevent or cure two inherited diseases in mice. In one study, researchers corrected the gene mutation that causes phenylketonuria (PKU) in the livers of adult mice; in the other, editing short-circuited a condition similar to the human disease HT1 in the livers of fetal mice.
In the PKU study, carried out at ETH Zurich in Switzerland, researchers bundled a CRISPR-Cas9 system plus the enzyme cytidine deaminase into an adeno-associated viral vector and injected it into the livers of mice with defective genes for phenylalanine hydroxylase. (A lack of functional phenylalanine hydroxylase causes a toxic buildup of phenylalanine in patients with PKU unless they’re diagnosed soon after birth and put on a special diet.) The gene-editing homed in on and changed a single base pair in the genomes of the ...
