Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood. Currently, there is no effective treatment or cure for MSMDS. A single error in the genetic code of the ACTA2 gene, which encodes the smooth muscle actin protein, is the most common cause of MSMDS. To directly target this mutation, researchers from Mass General Brigham engineered a bespoke CRISPR-Cas9 gene-editing enzyme to develop a potential therapy for MSMDS, which substantially prolonged survival and reduced vascular disease and neurodegeneration in mouse models of MSMDS. Findings are published in Nature Biomedical Engineering.

In a new study tracking the movement of fluorescent particles inside the cells of microscopic worms shows that the cytoplasm inside the worms was significantly more crowded and compartmentalized than in single-celled yeast or mammalian tissue culture cells, which are more commonly used to gauge internal cellular dynamics. The difference highlights the importance of studying cellular processes in living animals rather than cell culture.

A study supported by the National Institutes of Health (NIH) finds that individuals who received medication for opioid use disorder (MOUD) while incarcerated were significantly more likely to continue treatment six months after release than those who did not receive MOUD. The study also found that receiving MOUD in jail was associated with a 52% lower risk of fatal opioid overdose, a 24% lower risk of non-fatal opioid overdose, a 56% lower risk of death from any cause, and a 12% lower risk of reincarceration after release. These outcomes underscore the importance of providing MOUD treatment during incarceration.