Investigation of the pentamidine efficacy against myotonic dystrophy type 1 exploiting polymeric- and lipid-based nanoparticles.
Myotonic dystrophy type 1 (DM1) is a genetic disorder caused by a mutation in the DMPK gene, in which an expanded CTG trinucleotide is transcribed into abnormal mRNA that accumulates in nuclear foci. These foci sequester crucial splicing factors, causing a strong mis-regulation of downstream target genes. Consequently,...