Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise in correcting frame-disrupting mutations in the DMD gene for Duchenne muscular dystrophy. However, insufficient systemic delivery limits clinical adoption. Previously, we showed that a glucose/fructose mixture augmented AO delivery to muscle in mdx mice. Here, we evaluated if fructose alone could enhance...
Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise in correcting frame-disrupting mutations in the DMD gene for Duchenne muscular dystrophy. However, insufficient systemic delivery limits clinical adoption. Previously, we showed that a glucose/fructose mixture augmented AO delivery to muscle in mdx mice. Here, we evaluated if fructose alone could enhance...
We have recently reported that cell-penetrating peptides (CPPs) and novel chimeric peptides containing CPP (referred as
B peptide) and muscle-targeting peptide (referred as MSP) motifs significantly improve the systemic exon-skipping activity
of morpholino phosphorodiamidate oligomers (PMOs) in dystrophin-deficient mdx mice. In the present study, the general
mechanistic significance of the...
We have recently reported that cell-penetrating peptides (CPPs) and novel chimeric peptides containing CPP (referred as
B peptide) and muscle-targeting peptide (referred as MSP) motifs significantly improve the systemic exon-skipping activity
of morpholino phosphorodiamidate oligomers (PMOs) in dystrophin-deficient mdx mice. In the present study, the general
mechanistic significance of the...
Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise in correcting frame-disrupting mutations in the DMD gene for Duchenne muscular dystrophy. However, insufficient systemic delivery limits clinical adoption. Previously, we showed that a glucose/fructose mixture augmented AO delivery to muscle in mdx mice. Here, we evaluated if fructose alone could enhance...
We have recently reported that cell-penetrating peptides (CPPs) and novel chimeric peptides containing CPP (referred as
B peptide) and muscle-targeting peptide (referred as MSP) motifs significantly improve the systemic exon-skipping activity
of morpholino phosphorodiamidate oligomers (PMOs) in dystrophin-deficient mdx mice. In the present study, the general
mechanistic significance of the...