Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

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

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

Load More

Include forthcoming?