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

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

AR cooperates with SMAD4 to maintain skeletal muscle homeostasis.

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

Dystrophin involvement in peripheral circadian SRF signalling.

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

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

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

Plasma pNfH levels differentiate SBMA from ALS.

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

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

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

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

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

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

Cognitive decline in Huntington's disease expansion gene carriers.

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

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