Literature DB >> 20134472

Muscling in: Gene therapies for muscular dystrophy target RNA.

Joel R Chamberlain1, Jeffrey S Chamberlain.   

Abstract

Muscle diseases can take many forms, from the progressive muscle degeneration of dystrophies to the childhood cancer rhabdomyosarcoma. In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to Bench', Simone Hettmer and Amy J. Wagers examine the implications of clinical studies describing a type of rhabdomyosarcoma that resembles acute leukemia. The findings dovetail with other studies suggesting that some of these cancers might originate outside of muscle tissue and highlight the need for a better understanding of the cells that give rise to this condition.

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Year:  2010        PMID: 20134472     DOI: 10.1038/nm0210-170

Source DB:  PubMed          Journal:  Nat Med        ISSN: 1078-8956            Impact factor:   53.440


  14 in total

1.  DM2 intronic expansions: evidence for CCUG accumulation without flanking sequence or effects on ZNF9 mRNA processing or protein expression.

Authors:  Jamie M Margolis; Benedikt G Schoser; Melinda L Moseley; John W Day; Laura P W Ranum
Journal:  Hum Mol Genet       Date:  2006-04-19       Impact factor: 6.150

2.  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

3.  Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy.

Authors:  J W Miller; C R Urbinati; P Teng-Umnuay; M G Stenberg; B J Byrne; C A Thornton; M S Swanson
Journal:  EMBO J       Date:  2000-09-01       Impact factor: 11.598

4.  Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology.

Authors:  Julia Alter; Fang Lou; Adam Rabinowitz; HaiFang Yin; Jeffrey Rosenfeld; Steve D Wilton; Terence A Partridge; Qi Long Lu
Journal:  Nat Med       Date:  2006-01-29       Impact factor: 53.440

5.  Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

Authors:  Thurman M Wheeler; Krzysztof Sobczak; John D Lueck; Robert J Osborne; Xiaoyan Lin; Robert T Dirksen; Charles A Thornton
Journal:  Science       Date:  2009-07-17       Impact factor: 47.728

6.  Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.

Authors:  Aurélie Goyenvalle; Arran Babbs; Gert-Jan B van Ommen; Luis Garcia; Kay E Davies
Journal:  Mol Ther       Date:  2009-05-19       Impact factor: 11.454

7.  RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

Authors:  Saovaros Svasti; Thipparat Suwanmanee; Suthat Fucharoen; Hong M Moulton; Michelle H Nelson; Nobuyo Maeda; Oliver Smithies; Ryszard Kole
Journal:  Proc Natl Acad Sci U S A       Date:  2009-01-21       Impact factor: 11.205

8.  Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse.

Authors:  C J Mann; K Honeyman; A J Cheng; T Ly; F Lloyd; S Fletcher; J E Morgan; T A Partridge; S D Wilton
Journal:  Proc Natl Acad Sci U S A       Date:  2001-01-02       Impact factor: 11.205

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.  RNA toxicity is a component of ataxin-3 degeneration in Drosophila.

Authors:  Ling-Bo Li; Zhenming Yu; Xiuyin Teng; Nancy M Bonini
Journal:  Nature       Date:  2008-04-30       Impact factor: 49.962

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  5 in total

Review 1.  Animal models of muscular dystrophy.

Authors:  Rainer Ng; Glen B Banks; John K Hall; Lindsey A Muir; Julian N Ramos; Jacqueline Wicki; Guy L Odom; Patryk Konieczny; Jane Seto; Joel R Chamberlain; Jeffrey S Chamberlain
Journal:  Prog Mol Biol Transl Sci       Date:  2012       Impact factor: 3.622

Review 2.  Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors.

Authors:  Jane T Seto; Julian N Ramos; Lindsey Muir; Jeffrey S Chamberlain; Guy L Odom
Journal:  Curr Gene Ther       Date:  2012-06       Impact factor: 4.391

Review 3.  Therapeutics development in myotonic dystrophy type 1.

Authors:  Erin Pennock Foff; Mani S Mahadevan
Journal:  Muscle Nerve       Date:  2011-05-23       Impact factor: 3.217

4.  Systemic delivery of human mesenchymal stromal cells combined with IGF-1 enhances muscle functional recovery in LAMA2 dy/2j dystrophic mice.

Authors:  Mariane Secco; Carlos Bueno; Natassia M Vieira; Camila Almeida; Mayra Pelatti; Eder Zucconi; Paolo Bartolini; Mariz Vainzof; Elen H Miyabara; Oswaldo K Okamoto; Mayana Zatz
Journal:  Stem Cell Rev Rep       Date:  2013-02       Impact factor: 5.739

Review 5.  Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain.

Authors:  Brandon J Walters; Amber B Azam; Colleen J Gillon; Sheena A Josselyn; Iva B Zovkic
Journal:  Front Genet       Date:  2016-01-12       Impact factor: 4.599

  5 in total

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