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Publications

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

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.

Blain AM. et al, (2018), PLoS One, 13

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

Splice-Switching Therapy for Spinal Muscular Atrophy.

Meijboom KE. et al, (2017), Genes (Basel), 8

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

Identification of novel therapy-responsive protein biomarkers for Duchenne muscular dystrophy by aptamer-based serum proteomics

Coenen-Stass A. et al, (2015), NEUROMUSCULAR DISORDERS, 25, S251 - S252

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

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