Literature DB >> 21121926

New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.

Annemieke Aartsma-Rus1, Johan T den Dunnen, Gert-Jan B van Ommen.   

Abstract

The two therapeutic approaches currently most advanced in clinical trials for Duchenne muscular dystrophy are antisense-mediated exon skipping and forced read-through of premature stop codons. Interestingly, these approaches target the gene product rather than the gene itself. This review will explain the rationale and current state of affairs of these approaches and will then discuss how these gene-derived therapies might also be applicable to other diseases.
© 2010 New York Academy of Sciences.

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Year:  2010        PMID： 21121926     DOI： 10.1111/j.1749-6632.2010.05836.x

Source DB:  PubMed          Journal:  Ann N Y Acad Sci        ISSN： 0077-8923            Impact factor:   5.691


  9 in total

1.  [Neuromuscular diseases].

Authors:  B Schoser; S Zierz
Journal:  Nervenarzt       Date:  2011-06       Impact factor: 1.214

2.  Megalencephalic leukoencephalopathy with subcortical cysts type 1 (MLC1) due to a homozygous deep intronic splicing mutation (c.895-226T>G) abrogated in vitro using an antisense morpholino oligonucleotide.

Authors:  Cecilia Mancini; Giovanna Vaula; Laura Scalzitti; Simona Cavalieri; Enrico Bertini; Chiara Aiello; Cinzia Lucchini; Richard A Gatti; Alessandro Brussino; Alfredo Brusco
Journal:  Neurogenetics       Date:  2012-05-03       Impact factor: 2.660

3.  Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.

Authors:  Zejing Wang; Rainer Storb; Christine L Halbert; Glen B Banks; Tiffany M Butts; Eric E Finn; James M Allen; A Dusty Miller; Jeffrey S Chamberlain; Stephen J Tapscott
Journal:  Mol Ther       Date:  2012-06-12       Impact factor: 11.454

4.  Deep-intronic ATM mutation detected by genomic resequencing and corrected in vitro by antisense morpholino oligonucleotide (AMO).

Authors:  Simona Cavalieri; Elisa Pozzi; Richard A Gatti; Alfredo Brusco
Journal:  Eur J Hum Genet       Date:  2012-12-05       Impact factor: 4.246

5.  In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2.

Authors:  Elisabeth Castellanos; Imma Rosas; Ares Solanes; Isabel Bielsa; Conxi Lázaro; Cristina Carrato; Cristina Hostalot; Pepe Prades; Francesc Roca-Ribas; Ignacio Blanco; Eduard Serra
Journal:  Eur J Hum Genet       Date:  2012-11-28       Impact factor: 4.246

6.  Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations.

Authors:  Emily Dick; Spandan Kalra; David Anderson; Vinoj George; Morten Ritso; Steven H Laval; Rita Barresi; Annemieke Aartsma-Rus; Hanns Lochmüller; Chris Denning
Journal:  Stem Cells Dev       Date:  2013-07-05       Impact factor: 3.272

7.  The effects of low levels of dystrophin on mouse muscle function and pathology.

Authors:  Maaike van Putten; Margriet Hulsker; Vishna Devi Nadarajah; Sandra H van Heiningen; Ella van Huizen; Maarten van Iterson; Peter Admiraal; Tobias Messemaker; Johan T den Dunnen; Peter A C 't Hoen; Annemieke Aartsma-Rus
Journal:  PLoS One       Date:  2012-02-16       Impact factor: 3.240

Review 8.  Splicing therapy for neuromuscular disease.

Authors:  Andrew G L Douglas; Matthew J A Wood
Journal:  Mol Cell Neurosci       Date:  2013-04-28       Impact factor: 4.314

9.  6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes.

Authors:  Marika Pane; Elena S Mazzone; Maria Pia Sormani; Sonia Messina; Gian Luca Vita; Lavinia Fanelli; Angela Berardinelli; Yvan Torrente; Adele D'Amico; Valentina Lanzillotta; Emanuela Viggiano; Paola D'Ambrosio; Filippo Cavallaro; Silvia Frosini; Luca Bello; Serena Bonfiglio; Roberta Scalise; Roberto De Sanctis; Enrica Rolle; Flaviana Bianco; Marlene Van der Haawue; Francesca Magri; Concetta Palermo; Francesca Rossi; Maria Alice Donati; Chiara Alfonsi; Michele Sacchini; Maria Teresa Arnoldi; Giovanni Baranello; Tiziana Mongini; Antonella Pini; Roberta Battini; Elena Pegoraro; Stefano C Previtali; Sara Napolitano; Claudio Bruno; Luisa Politano; Giacomo P Comi; Enrico Bertini; Lucia Morandi; Francesca Gualandi; Alessandra Ferlini; Nathalie Goemans; Eugenio Mercuri
Journal:  PLoS One       Date:  2014-01-08       Impact factor: 3.240
  9 in total

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