CrisPR/CRIS systems are highly effective and useful for genomic manipulation. Despite this, cardiac treatment remains difficult due to existing genome editing and delivery processes.
Moataz Dowaidar
Abstract
CRISPR/Cas9 systems, in particular, have emerged as an important and promising technology for in vitro and in vivo genomic manipulation. Although genome editing has been used in the cardiovascular community as a scientific tool to study disease pathogenesis, therapeutic genome editing has yet to be used in clinical practice. PCSK9 is one of the most promising therapeutic genome editing candidates in the liver, and it has been thoroughly studied for cardiovascular disease risk reduction. Despite this, owing to the inefficiency of current genome editing technologies and distribution systems in the heart, treating the heart remains challenging. We should expect a steady stream of new editing approaches targeting wider use, given that clinical genome editing is still in its early stages but is rapidly progressing. It would be necessary to develop safe and reliable distribution systems in order to use such creative approaches in clinical settings.