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Publications

PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice.

Kordala AJ. et al, (2023), EMBO Mol Med, 15

A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles

Garcia-Guerra A. et al, (2023), Nanoscale Advances

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

MicroRNA-298 reduces levels of human amyloid-β precursor protein (APP), β-site APP-converting enzyme 1 (BACE1) and specific tau protein moieties.

Chopra N. et al, (2021), Mol Psychiatry, 26, 5636 - 5657

Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity.

Lim WF. et al, (2021), Sci Adv, 7

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

Variants in ATP6V0A1 cause progressive myoclonus epilepsy and developmental and epileptic encephalopathy.

Bott LC. et al, (2021), Brain Commun, 3

Plasma pNfH levels differentiate SBMA from ALS.

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

Polyglutamine-Expanded Androgen Receptor Alteration of Skeletal Muscle Homeostasis and Myonuclear Aggregation Are Affected by Sex, Age and Muscle Metabolism.

Chivet M. et al, (2020), Cells, 9

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

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

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

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

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents.

Pourshafie N. et al, (2018), J Vis Exp

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

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?

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

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

Cognitive decline in Huntington's disease expansion gene carriers.

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

The Multiple Faces of Spinocerebellar Ataxia type 2.

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

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