Literature DB >> 25198047

Activating internal ribosome entry to treat Duchenne muscular dystrophy.

Shireen R Lamandé1, Kathryn N North1.   

Abstract

Mutations in the DMD gene, encoding dystrophin, cause the most common forms of muscular dystrophy. A new study shows that forcing translation of DMD from an internal ribosome entry site can alleviate Duchenne muscular dystrophy symptoms in a mouse model.

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Year:  2014        PMID： 25198047     DOI： 10.1038/nm.3677

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


  10 in total

1.  DMD exon 1 truncating point mutations: amelioration of phenotype by alternative translation initiation in exon 6.

Authors:  Olga L Gurvich; Baijayanta Maiti; Robert B Weiss; Gaurav Aggarwal; Michael T Howard; Kevin M Flanigan
Journal:  Hum Mutat       Date:  2009-04       Impact factor: 4.878

2.  Becker muscular dystrophy with widespread muscle hypertrophy and a non-sense mutation of exon 2.

Authors:  N Witting; M Duno; J Vissing
Journal:  Neuromuscul Disord       Date:  2012-08-28       Impact factor: 4.296

3.  Second cistron in CACNA1A gene encodes a transcription factor mediating cerebellar development and SCA6.

Authors:  Xiaofei Du; Jun Wang; Haipeng Zhu; Lorenzo Rinaldo; Kay-Marie Lamar; Ann C Palmenberg; Christian Hansel; Christopher M Gomez
Journal:  Cell       Date:  2013-07-03       Impact factor: 41.582

4.  Attenuated APC alleles produce functional protein from internal translation initiation.

Authors:  Kathleen Heppner Goss; Chris Trzepacz; Thérèse M F Tuohy; Joanna Groden
Journal:  Proc Natl Acad Sci U S A       Date:  2002-05-28       Impact factor: 11.205

5.  Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.

Authors:  Aurélie Goyenvalle; Jordan Wright; Arran Babbs; Vivienne Wilkins; Luis Garcia; Kay E Davies
Journal:  Mol Ther       Date:  2012-02-21       Impact factor: 11.454

Review 6.  The current status of vertebrate cellular mRNA IRESs.

Authors:  Richard J Jackson
Journal:  Cold Spring Harb Perspect Biol       Date:  2013-02-01       Impact factor: 10.005

7.  An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus.

Authors:  A P Monaco; C J Bertelson; S Liechti-Gallati; H Moser; L M Kunkel
Journal:  Genomics       Date:  1988-01       Impact factor: 5.736

Review 8.  Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: progress and challenges.

Authors:  Virginia Arechavala-Gomeza; Karen Anthony; Jennifer Morgan; Francesco Muntoni
Journal:  Curr Gene Ther       Date:  2012-06       Impact factor: 4.391

9.  Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy.

Authors:  Aurélie Goyenvalle; Kay E Davies
Journal:  Skelet Muscle       Date:  2011-02-09       Impact factor: 4.912

10.  Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Authors:  Nicolas Wein; Adeline Vulin; Maria S Falzarano; Christina Al-Khalili Szigyarto; Baijayanta Maiti; Andrew Findlay; Kristin N Heller; Mathias Uhlén; Baskar Bakthavachalu; Sonia Messina; Giuseppe Vita; Chiara Passarelli; Simona Brioschi; Matteo Bovolenta; Marcella Neri; Francesca Gualandi; Steve D Wilton; Louise R Rodino-Klapac; Lin Yang; Diane M Dunn; Daniel R Schoenberg; Robert B Weiss; Michael T Howard; Alessandra Ferlini; Kevin M Flanigan
Journal:  Nat Med       Date:  2014-08-10       Impact factor: 53.440
  10 in total
  1 in total

1.  Therapeutic Effect of Losartan, an Angiotensin II Type 1 Receptor Antagonist, on CCl₄-Induced Skeletal Muscle Injury.

Authors:  Ok-Kyung Hwang; Jin-Kyu Park; Eun-Joo Lee; Eun-Mi Lee; Ah-Young Kim; Kyu-Shik Jeong
Journal:  Int J Mol Sci       Date:  2016-02-08       Impact factor: 5.923
  1 in total

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