Literature DB >> 25365985

Could exon skipping help dystrophic boys to run, hop, and jump?

Terence Partridge1.   

Abstract

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Year:  2014        PMID： 25365985      PMCID： PMC4429745          DOI： 10.1038/mt.2014.189

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  19 in total

1.  Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs.

Authors:  Toshifumi Yokota; Qi-Long Lu; Terence Partridge; Masanori Kobayashi; Akinori Nakamura; Shińichi Takeda; Eric Hoffman
Journal:  Ann Neurol       Date:  2009-06       Impact factor: 10.422

2.  Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Authors:  Nathalie M Goemans; Mar Tulinius; Johanna T van den Akker; Brigitte E Burm; Peter F Ekhart; Niki Heuvelmans; Tjadine Holling; Anneke A Janson; Gerard J Platenburg; Jessica A Sipkens; J M Ad Sitsen; Annemieke Aartsma-Rus; Gert-Jan B van Ommen; Gunnar Buyse; Niklas Darin; Jan J Verschuuren; Giles V Campion; Sjef J de Kimpe; Judith C van Deutekom
Journal:  N Engl J Med       Date:  2011-03-23       Impact factor: 91.245

3.  Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect.

Authors:  Kevin M Flanigan; Katie Campbell; Laurence Viollet; Wei Wang; Ana Maria Gomez; Christopher M Walker; Jerry R Mendell
Journal:  Hum Gene Ther       Date:  2013-09       Impact factor: 5.695

4.  One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.

Authors:  Bo Wu; Bin Xiao; Caryn Cloer; Mona Shaban; Arpana Sali; Peijuan Lu; Juan Li; Kanneboyina Nagaraju; Xiao Xiao; Qi Long Lu
Journal:  Mol Ther       Date:  2010-12-21       Impact factor: 11.454

5.  Eteplirsen for the treatment of Duchenne muscular dystrophy.

Authors:  Jerry R Mendell; Louise R Rodino-Klapac; Zarife Sahenk; Kandice Roush; Loren Bird; Linda P Lowes; Lindsay Alfano; Ann Maria Gomez; Sarah Lewis; Janaiah Kota; Vinod Malik; Kim Shontz; Christopher M Walker; Kevin M Flanigan; Marco Corridore; John R Kean; Hugh D Allen; Chris Shilling; Kathleen R Melia; Peter Sazani; Jay B Saoud; Edward M Kaye
Journal:  Ann Neurol       Date:  2013-09-10       Impact factor: 10.422

6.  Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.

Authors:  Aurélie Goyenvalle; Adeline Vulin; Françoise Fougerousse; France Leturcq; Jean-Claude Kaplan; Luis Garcia; Olivier Danos
Journal:  Science       Date:  2004-11-04       Impact factor: 47.728

7.  Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

Authors:  Adeline Vulin; Inès Barthélémy; Aurélie Goyenvalle; Jean-Laurent Thibaud; Cyriaque Beley; Graziella Griffith; Rachid Benchaouir; Maëva le Hir; Yves Unterfinger; Stéphanie Lorain; Patrick Dreyfus; Thomas Voit; Pierre Carlier; Stéphane Blot; Luis Garcia
Journal:  Mol Ther       Date:  2012-09-11       Impact factor: 11.454

8.  Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Authors:  Caroline Le Guiner; Marie Montus; Laurent Servais; Yan Cherel; Virginie Francois; Jean-Laurent Thibaud; Claire Wary; Béatrice Matot; Thibaut Larcher; Lydie Guigand; Maeva Dutilleul; Claire Domenger; Marine Allais; Maud Beuvin; Amélie Moraux; Johanne Le Duff; Marie Devaux; Nicolas Jaulin; Mickaël Guilbaud; Virginie Latournerie; Philippe Veron; Sylvie Boutin; Christian Leborgne; Diana Desgue; Jack-Yves Deschamps; Sophie Moullec; Yves Fromes; Adeline Vulin; Richard H Smith; Nicolas Laroudie; Frédéric Barnay-Toutain; Christel Rivière; Stéphanie Bucher; Thanh-Hoa Le; Nicolas Delaunay; Mehdi Gasmi; Robert M Kotin; Gisèle Bonne; Oumeya Adjali; Carole Masurier; Jean-Yves Hogrel; Pierre Carlier; Philippe Moullier; Thomas Voit
Journal:  Mol Ther       Date:  2014-08-04       Impact factor: 11.454

9.  Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.

Authors:  Maria Kinali; Virginia Arechavala-Gomeza; Lucy Feng; Sebahattin Cirak; David Hunt; Carl Adkin; Michela Guglieri; Emma Ashton; Stephen Abbs; Petros Nihoyannopoulos; Maria Elena Garralda; Mary Rutherford; Caroline McCulley; Linda Popplewell; Ian R Graham; George Dickson; Matthew J A Wood; Dominic J Wells; Steve D Wilton; Ryszard Kole; Volker Straub; Kate Bushby; Caroline Sewry; Jennifer E Morgan; Francesco Muntoni
Journal:  Lancet Neurol       Date:  2009-08-25       Impact factor: 44.182

10.  Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.

Authors:  Karen Anthony; Virginia Arechavala-Gomeza; Valeria Ricotti; Silvia Torelli; Lucy Feng; Narinder Janghra; Giorgio Tasca; Michela Guglieri; Rita Barresi; Annarita Armaroli; Alessandra Ferlini; Katherine Bushby; Volker Straub; Enzo Ricci; Caroline Sewry; Jennifer Morgan; Francesco Muntoni
Journal:  JAMA Neurol       Date:  2014-01       Impact factor: 18.302
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