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Do genome-editing technologies have a role in managing atherogenic dyslipidaemia?

A state-of-the art review suggests potential for novel genome-editing techniques, such as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR-associated 9), which make it feasible to permanently alter target genes in vivo. CRISPR-Cas9 may offer specific advantages over other genome-editing technologies, as this can be redirected to any genomic site. Indeed, in several proof-of-concept studies, permanent disruption of PCSK9 with genome editing was achieved in an in vivo mouse model. Future gene targets for this approach may include ANGPTL3 and APOC3. However, before this approach can be translated to humans, a number of issues need to be addressed. First, the precision of the technique needs to be improved. Additionally, the potential for unintended consequences at the target site such as insertion of undesired sequences, large deletions, and chromosomal rearrangements, off-target mutagenesis, and efficacy and safety need to be assessed. If proven to be effective and safe, genome-editing therapies could offer advantages for long-term management of atherogenic dyslipidaemia.
CRISPR-Cas9 Genome editing for treatment of atherogenic dyslipidemia.

Chadwick AC, Musunuru K.