Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Klein et. al., The Journal of Clinical Investigation (2019), online publication ahead of print version
Publication
Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
Gait et. al., Nucleic Acid Therapeutics (2018), 29(1), 1
How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse
Godfrey et. al., Human Molecular Genetics (2015), 24(15), 4225
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
Betts et. al., Molecular Therapy–Nucleic Acids (2012, 1(8), e38
Targeting RNA to treat neuromuscular disease
Muntoni and Woods, Nature Reviews Drug Discovery (2011), 10, 612