A blog for humans in the universe, to understand what we know now and to imagine what we don't know. To bridge the gap between the real world, its image in our brain and the ideas generated by.
samedi 23 avril 2016
Disruption of drug business? The one shot treatment of PCSK9 for lowering LDLc by CRISPR-Cas9
http://www.nature.com/nature/journal/v520/n7546/abs/nature14299.html
The RNA-guided endonuclease CRISPR-Cas9 is being widely adopted as the genome-editing tool of choice. However, the physical size of the commonly used Cas9 from Streptococcus pyogenes (SpCas9) poses problems for applications that use the adeno-associated virus (AAV) as a delivery vehicle. Feng Zhang and colleagues now characterize six smaller Cas9 orthologues. Focusing on Cas9 fromStaphylococcus aureus, they packaged it and its single guide RNA expression cassette into a single AAV vector and targeted the cholesterol regulatory gene Pcsk9 in the mouse liver. Greater than 40%gene modification was observed within a week of injection, accompanied by significant reductions in serum Pcsk9 and total cholesterol levels.
The RNA-guided endonuclease CRISPR-Cas9 is being widely adopted as the genome-editing tool of choice. However, the physical size of the commonly used Cas9 from Streptococcus pyogenes (SpCas9) poses problems for applications that use the adeno-associated virus (AAV) as a delivery vehicle. Feng Zhang and colleagues now characterize six smaller Cas9 orthologues. Focusing on Cas9 fromStaphylococcus aureus, they packaged it and its single guide RNA expression cassette into a single AAV vector and targeted the cholesterol regulatory gene Pcsk9 in the mouse liver. Greater than 40%gene modification was observed within a week of injection, accompanied by significant reductions in serum Pcsk9 and total cholesterol levels.