Skip to main content
Premium Trial:

Request an Annual Quote

This Week in Science: Jun 30, 2017

In this week's Science Translational Medicine, an international research team reports the identification of two proteins that may help treat muscular dystrophy. Nearly half of all cases of the disease are caused by mutations in the LAMA2 gene, but gene replacement therapies have been ineffective for these cases because of the amount of proteins required to restore muscle functionality. To address this, the investigators genetically engineered a mouse model of muscular dystrophy to express two smaller proteins, which were able to reverse the effects of the disease by acting as linkers that corrected defective connections between muscle fibers and the basement membrane.

Also in Science Translational Medicine, a multi-institute team of researchers describes the discovery of a potential tumor suppressor that may be behind drug resistance in follicular lymphomas (FLs). The investigators focused on the 6q region of chromosome 6, which is missing large portions in about a third of all FLs and screened 81 genes for their ability to promote cellular proliferation. One in particular — SESTRIN1 — was identified as a tumor suppressor, and its downregulation in mice led to accelerated FL development. Notably, tumors lacking SESTRIN1 were resistant to treatment with an experimental lymphoma therapy that was found to target an activator of SESTRIN1 called EZH2. The results suggest that SESTRIN1 and EZH2 mutations should be considered in the development of lymphoma therapies.