Publications
Key publications
Antisense oligonucleotides: the next frontier for treatment of neurological disorders.
Rinaldi C. and Wood MJA., (2018), Nat Rev Neurol, 14, 9 - 21
C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia.
Aoki Y. et al, (2017), Brain, 140, 887 - 897
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
Hammond SM. et al, (2016), Proc Natl Acad Sci U S A, 113, 10962 - 10967
Exosomes surf on filopodia to enter cells at endocytic hot spots, traffic within endosomes, and are targeted to the ER.
Heusermann W. et al, (2016), J Cell Biol, 213, 173 - 184
Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.
Han G. et al, (2016), Nat Commun, 7
Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides.
Ezzat K. et al, (2015), Nano Lett, 15, 4364 - 4373
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.
Goyenvalle A. et al, (2015), Nat Med, 21, 270 - 275
Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model.
Bestas B. et al, (2014), J Clin Invest, 124, 4067 - 4081
Extracellular vesicles: biology and emerging therapeutic opportunities.
EL Andaloussi S. et al, (2013), Nat Rev Drug Discov, 12, 347 - 357
Exosome-mediated delivery of siRNA in vitro and in vivo.
El-Andaloussi S. et al, (2012), Nat Protoc, 7, 2112 - 2126
Targeting RNA to treat neuromuscular disease.
Muntoni F. and Wood MJA., (2011), Nat Rev Drug Discov, 10, 621 - 637
Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes.
Alvarez-Erviti L. et al, (2011), Nat Biotechnol, 29, 341 - 345
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.
Kinali M. et al, (2009), Lancet Neurol, 8, 918 - 928
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.
Yin H. et al, (2008), Hum Mol Genet, 17, 3909 - 3918
Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle.
Ivanova GD. et al, (2008), Nucleic Acids Res, 36, 6418 - 6428
Ribozyme-mediated trans-splicing of a trinucleotide repeat.
Phylactou LA. et al, (1998), Nat Genet, 18, 378 - 381