Feasibility is demonstrated in mammalian models for a therapeutic strategy that increases MBNL by sequestering MBNL-suppressive miRs.
Insights on the mechanisms that underlie muscle atrophy in DM are provided by studies in a new mouse model.
The RNA binding protein, rbFOX1, competes with MBNL in binding to CCUGexp, but not CUGexp repeats, and thereby mitigates DM2.
The review journal, Frontiers in Neurology, is publishing six new articles on myotonic dystrophy.
Intronic GC-rich microsatellite expansions are common in neurological disorders and act to trigger the intronic retention that underlies disease pathogenesis.