Browsing: Editing

Rather than correcting the expanded CAG repeat that causes Huntington’s disease, the researchers edited the splice acceptor of HTT exon 13. This caused full or partial exon skipping. The partial event removed 39 bases while preserving the reading frame and eliminating a site where proteolysis can generate aggregation-prone N-terminal huntingtin fragments

The US government’s health research moonshot agency has launched a five-year, $160 million bet that the era of bespoke gene-editing cures — currently limited to children whose families can access a vast scientific team willing to absorb the cost — can be converted into an industrial platform available to the 30 million Americans living with…