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Delivery is key: lessons learnt from developing splice-switching antisense therapies.
Published in:
EMBO Molecular Medicine, 2017, v. 9, n. 5, p. 545, doi. 10.15252/emmm.201607199
By:
- Godfrey, Caroline;
- Desviat, Lourdes R;
- Smedsrød, Bård;
- Piétri‐Rouxel, France;
- Denti, Michela A;
- Disterer, Petra;
- Lorain, Stéphanie;
- Nogales‐Gadea, Gisela;
- Sardone, Valentina;
- Anwar, Rayan;
- EL Andaloussi, Samir;
- Lehto, Taavi;
- Khoo, Bernard;
- Brolin, Camilla;
- Roon‐Mom, Willeke MC;
- Goyenvalle, Aurélie;
- Aartsma‐Rus, Annemieke;
- Arechavala‐Gomeza, Virginia
Publication type:
Article
Retention of hexanucleotide repeatcontaining intron in C9orf72 mRNA: implications for the pathogenesis of ALS/FTD.
Published in:
Acta Neuropathologica Communications, 2016, v. 4, p. 1, doi. 10.1186/s40478-016-0289-4
By:
- Niblock, Michael;
- Smith, Bradley N.;
- Lee, Youn-Bok;
- Sardone, Valentina;
- Topp, Simon;
- Troakes, Claire;
- Al-Sarraj, Safa;
- Leblond, Claire S.;
- Dion, Patrick A.;
- Rouleau, Guy A.;
- Shaw, Christopher E.;
- Gallo, Jean-Marc
Publication type:
Article
Allele-Specific Knockdown of ALS-Associated Mutant TDP-43 in Neural Stem Cells Derived from Induced Pluripotent Stem Cells.
Published in:
PLoS ONE, 2014, v. 9, n. 3, p. 1, doi. 10.1371/journal.pone.0091269
By:
- Nishimura, Agnes L.;
- Shum, Carole;
- Scotter, Emma L.;
- Abdelgany, Amr;
- Sardone, Valentina;
- Wright, Jamie;
- Lee, Youn-Bok;
- Chen, Han-Jou;
- Bilican, Bilada;
- Carrasco, Monica;
- Maniatis, Tom;
- Chandran, Siddharthan;
- Rogelj, Boris;
- Gallo, Jean-Marc;
- Shaw, Christopher E.
Publication type:
Article
TUBA4A gene analysis in sporadic amyotrophic lateral sclerosis: identification of novel mutations.
Published in:
2015
By:
- Pensato, Viviana;
- Tiloca, Cinzia;
- Corrado, Lucia;
- Bertolin, Cinzia;
- Sardone, Valentina;
- Bo, Roberto;
- Calini, Daniela;
- Mandrioli, Jessica;
- Lauria, Giuseppe;
- Mazzini, Letizia;
- Querin, Giorgia;
- Ceroni, Mauro;
- Cantello, Roberto;
- Corti, Stefania;
- Castellotti, Barbara;
- Soldà, Giulia;
- Duga, Stefano;
- Comi, Giacomo;
- Cereda, Cristina;
- Sorarù, Gianni
Publication type:
Letter
Expanded G4C2 repeats linked to C9ORF72 ALS and FTD form length-dependent RNA foci, sequester RNA binding proteins and are neurotoxic.
Published in:
2013
By:
- Youn-Bok Lee;
- Han-Jou Chen;
- Peres, João N.;
- Gomez, Jorge;
- Sardone, Valentina;
- Nishimura, Agnes L.;
- Scotter, Emma;
- Vance, Caroline;
- Štalekar, Maja;
- Adachi, Yoshitsugu;
- Troakes, Claire;
- Miller, Jack;
- Smith, Bradley;
- Hirth, Frank;
- Rogelj, Boris;
- Houart, Corinne;
- Shaw, Christopher E.
Publication type:
Abstract
Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.
Published in:
Molecules, 2017, v. 22, n. 4, p. 563, doi. 10.3390/molecules22040563
By:
- Sardone, Valentina;
- Haiyan Zhou;
- Muntoni, Francesco;
- Ferlini, Alessandra;
- Falzarano, Maria Sofia
Publication type:
Article
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
Published in:
PLoS ONE, 2018, v. 13, n. 10, p. 1, doi. 10.1371/journal.pone.0204485
By:
- Hiller, Monika;
- Falzarano, Maria Sofia;
- Garcia-Jimenez, Iker;
- Sardone, Valentina;
- Verheul, Ruurd C.;
- Popplewell, Linda;
- Anthony, Karen;
- Ruiz-Del-Yerro, Estibaliz;
- Osman, Hana;
- Goeman, Jelle J.;
- Mamchaoui, Kamel;
- Dickson, George;
- Ferlini, Alessandra;
- Muntoni, Francesco;
- Aartsma-Rus, Annemieke;
- Arechavala-Gomeza, Virginia;
- Datson, Nicole A.;
- Spitali, Pietro
Publication type:
Article
From Transcriptome to Noncoding RNAs: Implications in ALS Mechanism.
Published in:
Neurology Research International, 2012, p. 1, doi. 10.1155/2012/278725
By:
- Gagliardi, Stella;
- Milani, Pamela;
- Sardone, Valentina;
- Pansarasa, Orietta;
- Cereda, Cristina
Publication type:
Article
A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples.
Published in:
PLoS ONE, 2018, v. 13, n. 3, p. 1, doi. 10.1371/journal.pone.0194540
By:
- Sardone, Valentina;
- Ellis, Matthew;
- Torelli, Silvia;
- Feng, Lucy;
- Chambers, Darren;
- Eastwood, Deborah;
- Sewry, Caroline;
- Phadke, Rahul;
- Morgan, Jennifer E.;
- Muntoni, Francesco
Publication type:
Article

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