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Search results (35)

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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

Peptides for nucleic acid delivery.

Lehto T. et al, (2016), Adv Drug Deliv Rev, 106, 172 - 182

Genomic Rearrangements Considered as Quantitative Traits

Imprialou M. et al, (2016)

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

Selective release of muscle-specific, extracellular microRNAs during myogenic differentiation.

Coenen-Stass AML. et al, (2016), Hum Mol Genet, 25, 3960 - 3974

The Amount of Mitochondrial DNA in Blood Reflects the Course of a Depressive Episode.

Nicod J. et al, (2016), Biol Psychiatry, 80, e41 - e42

WFS1 in Optic Neuropathies: Mutation Findings in Nonsyndromic Optic Atrophy and Assessment of Clinical Severity.

Grenier J. et al, (2016), Ophthalmology, 123, 1989 - 1998

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

Peccate C. et al, (2016), Hum Mol Genet, 25, 3555 - 3563

Genome-wide association of multiple complex traits in outbred mice by ultra-low-coverage sequencing.

Nicod J. et al, (2016), Nat Genet, 48, 912 - 918

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.

Straub V. et al, (2016), Lancet Neurol, 15, 882 - 890

Precision Modulation of Neurodegenerative Disease-Related Gene Expression in Human iPSC-Derived Neurons.

Heman-Ackah SM. et al, (2016), Sci Rep, 6

Extracellular vesicles in neurodegenerative disease - pathogenesis to biomarkers.

Thompson AG. et al, (2016), Nat Rev Neurol, 12, 346 - 357

Spinocerebellar ataxia type 7 in South Africa: Epidemiology, pathogenesis and therapy.

Watson L. et al, (2016), S Afr Med J, 106, S107 - S109

A small-molecule Nrf1 and Nrf2 activator mitigates polyglutamine toxicity in spinal and bulbar muscular atrophy.

Bott LC. et al, (2016), Hum Mol Genet, 25, 1979 - 1989

MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy.

Pourshafie N. et al, (2016), Mol Ther, 24, 937 - 945

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

Reduced expression of MyHC slow isoform in rat soleus during unloading is accompanied by alterations of endogenous inhibitors of calcineurin/NFAT signaling pathway

Lomonosova YN. et al, (2016), Journal of Muscle Research and Cell Motility, 37, 7 - 16

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.

Han G. et al, (2016), Nat Commun, 7

Lipid-based Transfection Reagents Exhibit Cryo-induced Increase in Transfection Efficiency.

Sork H. et al, (2016), Mol Ther Nucleic Acids, 5

Cells release subpopulations of exosomes with distinct molecular and biological properties.

Willms E. et al, (2016), Sci Rep, 6

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