Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics.
McClorey G. and Banerjee S., (2018), Biomedicines, 6
PROFILE OF CIRCADIANLY REGULATED METABOLIC GENES IN DYSTROPHIC HEART
Betts CA. et al, (2018), HEART, 104, A4 - A4
Preclinical studies of WVE-210201, an investigational stereopure antisense oligonucleotide in development for the treatment of patients with duchenne muscular dystrophy (DMD)
Panzara M. et al, (2017), JOURNAL OF THE NEUROLOGICAL SCIENCES, 381, 277 - 278
WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration
Wood M. et al, (2017), NEUROMUSCULAR DISORDERS, 27, S217 - S217
Corrigendum: Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics.
Coenen-Stass AML. et al, (2016), Sci Rep, 6
Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.
Peccate C. et al, (2016), Hum Mol Genet, 25, 3555 - 3563
Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration.
Roberts TC. et al, (2015), Hum Mol Genet, 24, 6756 - 6768
Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics.
Coenen-Stass AML. et al, (2015), Sci Rep, 5
An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies.
McClorey G. and Wood MJ., (2015), Curr Opin Pharmacol, 24, 52 - 58
The physiological consequences of different levels of dystrophin following antisense based exon-skipping in the mdx mouse
Muses S. et al, (2015), NEUROMUSCULAR DISORDERS, 25, S312 - S313
Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy
(2015), Nucleic Acids Research, 43, 29 - 39
Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies.
Rouillon J. et al, (2015), Hum Mol Genet, 24, 4916 - 4932
How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse.
Godfrey C. et al, (2015), Hum Mol Genet, 24, 4225 - 4237
Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides.
Ezzat K. et al, (2015), Nano Lett, 15, 4364 - 4373
Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy.
Betts CA. et al, (2015), Sci Rep, 5
Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides.
Järver P. et al, (2015), Nucleic Acid Ther, 25, 65 - 77
Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.
Betts CA. et al, (2015), Sci Rep, 5
Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.
Shabanpoor F. et al, (2015), Nucleic Acids Res, 43, 29 - 39