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.

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity.

Journal article

Kandasamy P. et al, (2022), Nucleic Acids Res, 50, 5443 - 5466

AR cooperates with SMAD4 to maintain skeletal muscle homeostasis.

Journal article

Forouhan M. et al, (2022), Acta Neuropathol, 143, 713 - 731

Dystrophin involvement in peripheral circadian SRF signalling.

Journal article

Betts CA. et al, (2021), Life Sci Alliance, 4

Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy.

Journal article

Winkelsas AM. et al, (2021), Mol Ther Nucleic Acids, 23, 731 - 742

Plasma pNfH levels differentiate SBMA from ALS.

Journal article

Lombardi V. et al, (2020), J Neurol Neurosurg Psychiatry, 91, 215 - 217

Muscle and not neuronal biomarkers correlate with severity in spinal and bulbar muscular atrophy.

Journal article

Lombardi V. et al, (2019), Neurology, 92, e1205 - e1211

Identification of potent, muscle-targeting investigational stereo-pure oligonucleotides for exon 53 DMD therapy

Conference paper

Durbin A. et al, (2018), NEUROMUSCULAR DISORDERS, 28, S67 - S67

Beyond motor neurons: expanding the clinical spectrum in Kennedy's disease.

Journal article

Manzano R. et al, (2018), J Neurol Neurosurg Psychiatry, 89, 808 - 812

From gene to therapy in spinal and bulbar muscular atrophy: Are we there yet?

Journal article

Pennuto M. and Rinaldi C., (2018), Mol Cell Endocrinol, 465, 113 - 121

Antisense oligonucleotides: the next frontier for treatment of neurological disorders.

Journal article

Rinaldi C. and Wood MJA., (2018), Nat Rev Neurol, 14, 9 - 21

Cognitive decline in Huntington's disease expansion gene carriers.

Journal article

Baake V. et al, (2017), Cortex, 95, 51 - 62

The Multiple Faces of Spinocerebellar Ataxia type 2.

Journal article

Antenora A. et al, (2017), Ann Clin Transl Neurol, 4, 687 - 695

Load More