V. Sardone

发表

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures

J. Goeman, F. Muntoni, N. Datson, 2018, PloS one.

Allele-Specific Knockdown of ALS-Associated Mutant TDP-43 in Neural Stem Cells Derived from Induced Pluripotent Stem Cells

T. Maniatis, C. Shaw, M. Carrasco, 2014, PloS one.

Hydrogen peroxide-mediated induction of SOD1 gene transcription is independent from Nrf2 in a cellular model of neurodegeneration.

M. Bordoni, O. Pansarasa, C. Cereda, 2016, Biochimica et biophysica acta.

Hydrogen peroxide-dependent oxidative stress induces SOD1 transcription gene is independent from Nrf2 transcription factor in a cellular model of neurodegeneration

M. Bordoni, O. Pansarasa, C. Cereda, 2015 .

Hexanucleotide Repeats in ALS/FTD Form Length-Dependent RNA Foci, Sequester RNA Binding Proteins, and Are Neurotoxic

J. Ule, C. Shaw, C. Troakes, 2013, Cell reports.

TUBA4A gene analysis in sporadic amyotrophic lateral sclerosis: identification of novel mutations

G. Comi, S. Corti, C. Shaw, 2015, Journal of Neurology.

Edinburgh Explorer Allele-Specific Knockdown of ALS-Associated Mutant TDP-43 in Neural Stem Cells Derived from Induced Pluripotent Stem Cells

T. Maniatis, C. Shaw, M. Carrasco, 2018 .

High-Throughput Digital Image Analysis Reveals Distinct Patterns of Dystrophin Expression in Dystrophinopathy Patients

F. Muntoni, V. Sardone, E. Ricci, 2021, Journal of neuropathology and experimental neurology.

OP-JNEN210096 1..11

F. Muntoni, V. Sardone, E. Ricci, 2021 .

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

E. Mercuri, F. Muntoni, L. Servais, 2020, Neurology.

A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples

F. Muntoni, V. Sardone, C. Sewry, 2018, PloS one.

Golodirsen Induces Exon Skipping Leading to Sarcolemmal Dystrophin Expression in Duchenne Muscular Dystrophy Patients With Mutations Amenable to Exon 53 Skipping (S22.001)

C. Clark, E. Mercuri, K. Bushby, 2018 .

From Transcriptome to Noncoding RNAs: Implications in ALS Mechanism

O. Pansarasa, V. Sardone, P. Milani, 2012, Neurology research international.

Differential roles of the ubiquitin proteasome system and autophagy in the clearance of soluble and aggregated TDP-43 species

D. Rubinsztein, C. Shaw, C. Vance, 2014, Journal of Cell Science.

Delivery is key: lessons learnt from developing splice‐switching antisense therapies

Samir El Andaloussi, Bård Smedsrød, B. Smedsrød, 2017, EMBO molecular medicine.

Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

F. Muntoni, M. Falzarano, V. Sardone, 2017, Molecules.

Retention of hexanucleotide repeat-containing intron in C9orf72 mRNA: implications for the pathogenesis of ALS/FTD

C. Shaw, C. Troakes, S. Al-Sarraj, 2016, Acta Neuropathologica Communications.

Posttranscriptional regulation of SOD1 gene expression under oxidative stress: Potential role of ELAV proteins in sporadic ALS

U. Laforenza, V. Sardone, S. Govoni, 2013, Neurobiology of Disease.

HuD regulates SOD1 expression during oxidative stress in differentiated neuroblastoma cells and sporadic ALS motor cortex

N. Perrone-Bizzozero, M. Bordoni, O. Pansarasa, 2020, Neurobiology of Disease.

Expanded G4C2 repeats linked to C9ORF72 ALS and FTD form length-dependent RNA foci, sequester RNA binding proteins and are neurotoxic

C. Shaw, C. Troakes, C. Vance, 2013, Molecular Neurodegeneration.