Literature DB >> 19713153

Less is more: therapeutic exon skipping for Duchenne muscular dystrophy.

Annemieke Aartsma-Rus, Gert-Jan B van Ommen.   

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Year:  2009        PMID： 19713153     DOI： 10.1016/S1474-4422(09)70229-7

Source DB:  PubMed          Journal:  Lancet Neurol        ISSN： 1474-4422            Impact factor:   44.182


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  12 in total

Review 1.  Silencing disease genes in the laboratory and the clinic.

Authors:  Jonathan K Watts; David R Corey
Journal:  J Pathol       Date:  2011-11-09       Impact factor: 7.996

Review 2.  MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction.

Authors:  Verena Behrens-Gawlik; Giulia Mearini; Christina Gedicke-Hornung; Pascale Richard; Lucie Carrier
Journal:  Pflugers Arch       Date:  2013-12-12       Impact factor: 3.657

3.  Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts.

Authors:  Pavithra S Iyer; Lionel O Mavoungou; Flavio Ronzoni; Joanna Zemla; Emanuel Schmid-Siegert; Stefania Antonini; Laurence A Neff; Olivier M Dorchies; Marisa Jaconi; Malgorzata Lekka; Graziella Messina; Nicolas Mermod
Journal:  Mol Ther       Date:  2018-02-02       Impact factor: 11.454

4.  Antisense Oligonucleotide Treatment in a Humanized Mouse Model of Duchenne Muscular Dystrophy and Highly Sensitive Detection of Dystrophin Using Western Blotting.

Authors:  Rika Maruyama; Toshifumi Yokota
Journal:  Methods Mol Biol       Date:  2021

5.  Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.

Authors:  Hans Heemskerk; Christa de Winter; Petra van Kuik; Niki Heuvelmans; Patrizia Sabatelli; Paola Rimessi; Paola Braghetta; Gert-Jan B van Ommen; Sjef de Kimpe; Alessandra Ferlini; Annemieke Aartsma-Rus; Judith C T van Deutekom
Journal:  Mol Ther       Date:  2010-04-20       Impact factor: 11.454

Review 6.  Genomics of alternative splicing: evolution, development and pathophysiology.

Authors:  Eric R Gamazon; Barbara E Stranger
Journal:  Hum Genet       Date:  2014-01-01       Impact factor: 4.132

7.  Chemical treatment enhances skipping of a mutated exon in the dystrophin gene.

Authors:  Atsushi Nishida; Naoyuki Kataoka; Yasuhiro Takeshima; Mariko Yagi; Hiroyuki Awano; Mitsunori Ota; Kyoko Itoh; Masatoshi Hagiwara; Masafumi Matsuo
Journal:  Nat Commun       Date:  2011       Impact factor: 14.919

Review 8.  Pharmacology of Modulators of Alternative Splicing.

Authors:  David O Bates; Jonathan C Morris; Sebastian Oltean; Lucy F Donaldson
Journal:  Pharmacol Rev       Date:  2017-01       Impact factor: 25.468

Review 9.  RNA Splicing: A New Paradigm in Host-Pathogen Interactions.

Authors:  Komal Chauhan; Haroon Kalam; Ravi Dutt; Dhiraj Kumar
Journal:  J Mol Biol       Date:  2019-03-08       Impact factor: 5.469

10.  Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method.

Authors:  Jean-Paul Iyombe-Engembe; Dominique L Ouellet; Xavier Barbeau; Joël Rousseau; Pierre Chapdelaine; Patrick Lagüe; Jacques P Tremblay
Journal:  Mol Ther Nucleic Acids       Date:  2016-01-26       Impact factor: 10.183
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