Key result
GFP-tagged MBNL protein co-localizes with DMPK expanded-repeat transcript foci in DM1 cell lines (p<0.01), whereas CUG-BP and hnRNP C do not, supporting a primary role for MBNL in DM1.
Population
DM1 cell lines and non-DM1 cell lines
Comparison
Indirect immunofluorescence and overexpression… vs Non-DM1 cell lines
Design
Preclinical
Authors
Loading...
Hypothesis-generating for MBNL-targeted DM1 therapies; leaves open in vivo efficacy and clinical translation.
p-value: p=<0.01
MBNL co-localises with expanded repeat transcripts in DM1 cells, supporting its involvement in the pathophysiology of myotonic dystrophy.
Majid Fardaei (2001) studied Myotonic dystrophy (DM1). GFP-tagged MBNL, CUG-BP, and hnRNP C transfection vs. Control fibroblast cell lines was evaluated on Co-localization of proteins with expanded repeat transcript foci (p=<0.01). GFP-tagged MBNL protein co-localizes with DMPK expanded-repeat transcript foci in DM1 cell lines (p<0.01), whereas CUG-BP and hnRNP C do not, supporting a primary role for MBNL in DM1.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: