The inventors of cryo-electron microscopy snapped up the Nobel Prize in Chemistry this month (see October 2017 news). The structural technique, pioneered in the mid-1970s, is emerging as a key approach to draft molecular blueprints of complex intracellular structures including the nuclear pore, which may clog up in ALS (see August 2015 news; for review, see Hoelz et al., 2016).
The strategy recently enabled Daniel Southworth’s team at the University of Michigan to capture molecular snapshots of Hsp104, an enzyme which helps refold aggregated proteins (Gates et al., 2017). The approach is currently being developed by University of Pennsylvania School of Medicine’s James Shorter as a potential therapy for ALS (see September 2017 news). Efforts to optimize this approach using these molecular blueprints, are now underway.
To learn more about how scientists aim to leverage existing enzymes to bust up inclusions in ALS, check out our recent feature: Breaking up TDP-43 aggregates may be doable.
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