Close monitoring of respiratory function reduces respiratory complications in DM1.
MDF has released a Request for Applications for projects that address the evaluation of genome editing strategies for DM1. The focus of this RFA is early stage discovery and development of in vivo genome editing technologies. Up to two awards of $250,000 each are planned.
NIAMS issues new funding opportunities that encourage innovative research—really!!
Recent studies have shown that MBNL exhibits differential dose-response relationships across the various gene translation events that it regulates in health and disease. It is as yet unclear precisely how the structure of the pre-mRNA itself contributes to patterning of MBNL-dependent alternative splicing.
Fuchs’ endothelial corneal dystrophy is mechanistically related to and commonly observed in DM1 families even in the absence of the Fuchs’ mutation.