The presence of cell and tissue barriers together with the low biomembrane permeability of
various therapeutics often hampers systemic drug distribution; thus, most of the available …

Here, we discuss three RNA-based therapeutic technologies exploiting various
oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have …

Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …

Recent studies have highlighted that splicing patterns are frequently altered in cancer and
that mutations in genes encoding spliceosomal proteins, as well as mutations affecting the …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Molecules that can specifically silence gene expression are powerful research tools. Much
effort has been put into the development of such molecules and has resulted in the creation …

Background Mutations that disrupt the open reading frame and prevent full translation of
DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Nonsense-mediated decay (NMD) eliminates mRNAs containing premature termination
codons and thus helps limit the synthesis of abnormal proteins. New results uncover a …

Background Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle
deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon …