The novel myosin ATPase inhibitor, Mavacamten, shows promising results in treating HOCM with noticeable symptomatic improvement! Fantastic study by Dr. Spertus, et. al.: thelancet.com/journals/lance…

Original paper in Science for the discovery of Mavacamten: pubmed.ncbi.nlm.nih.gov/26912705/

A neat method: SarcTrack. Use fluorescently labeled cardiac sarcomere to measure contraction.

Original paper using genomically tagged fluorescent protein: pubmed.ncbi.nlm.nih.gov/30700234/
More recent paper using virally transduced fluorescent protein: pubmed.ncbi.nlm.nih.gov/33557580/

Wonderful study with permanent genome editing in a liver-enriched fashion using lipid nanoparticles containing CRISPR base editors: nature.com/articles/s4158…. Really glad to see a lack of germline modification. As a #physicianscientist , this is very inspiring translational work!