Literature DB >> 25992360

Gene therapy in myotubular myopathy: promising progress and future directions.

Christopher R Pierson1.   

Abstract

Entities:  

Year:  2015        PMID： 25992360      PMCID： PMC4402603          DOI： 10.3978/j.issn.2305-5839.2015.01.11

Source DB:  PubMed          Journal:  Ann Transl Med        ISSN： 2305-5839


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  16 in total

1.  Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.

Authors:  Dawn E Bowles; Scott W J McPhee; Chengwen Li; Steven J Gray; Jade J Samulski; Angelique S Camp; Juan Li; Bing Wang; Paul E Monahan; Joseph E Rabinowitz; Joshua C Grieger; Lakshmanan Govindasamy; Mavis Agbandje-McKenna; Xiao Xiao; R Jude Samulski
Journal:  Mol Ther       Date:  2011-11-08       Impact factor: 11.454

2.  Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.

Authors:  Alice Toromanoff; Yan Chérel; Mickaël Guilbaud; Magalie Penaud-Budloo; Richard O Snyder; Mark E Haskins; Jack-Yves Deschamps; Lydie Guigand; Guillaume Podevin; Valder R Arruda; Katherine A High; Hansell H Stedman; Fabienne Rolling; Ignacio Anegon; Philippe Moullier; Caroline Le Guiner
Journal:  Mol Ther       Date:  2008-05-06       Impact factor: 11.454

3.  A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C.

Authors:  Serge Herson; Faycal Hentati; Aude Rigolet; Anthony Behin; Norma B Romero; France Leturcq; Pascal Laforêt; Thierry Maisonobe; Rim Amouri; Hafedh Haddad; Muriel Audit; Marie Montus; Carole Masurier; Bernard Gjata; Christophe Georger; Mustapha Cheraï; Pierre Carlier; Jean-Yves Hogrel; Ariane Herson; Yves Allenbach; François M Lemoine; David Klatzmann; H Lee Sweeney; Richard C Mulligan; Bruno Eymard; Didier Caizergues; Thomas Voït; Olivier Benveniste
Journal:  Brain       Date:  2012-01-11       Impact factor: 13.501

4.  A gene mutated in X-linked myotubular myopathy defines a new putative tyrosine phosphatase family conserved in yeast.

Authors:  J Laporte; L J Hu; C Kretz; J L Mandel; P Kioschis; J F Coy; S M Klauck; A Poustka; N Dahl
Journal:  Nat Genet       Date:  1996-06       Impact factor: 38.330

5.  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

6.  Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

Authors:  Paul Gregorevic; Michael J Blankinship; James M Allen; Jeffrey S Chamberlain
Journal:  Mol Ther       Date:  2008-03-04       Impact factor: 11.454

7.  The lipid phosphatase myotubularin is essential for skeletal muscle maintenance but not for myogenesis in mice.

Authors:  Anna Buj-Bello; Vincent Laugel; Nadia Messaddeq; Hala Zahreddine; Jocelyn Laporte; Jean-Francois Pellissier; Jean-Louis Mandel
Journal:  Proc Natl Acad Sci U S A       Date:  2002-10-21       Impact factor: 11.205

Review 8.  Centronuclear (myotubular) myopathy.

Authors:  Heinz Jungbluth; Carina Wallgren-Pettersson; Jocelyn Laporte
Journal:  Orphanet J Rare Dis       Date:  2008-09-25       Impact factor: 4.123

9.  Loss of myotubularin function results in T-tubule disorganization in zebrafish and human myotubular myopathy.

Authors:  James J Dowling; Andrew P Vreede; Sean E Low; Elizabeth M Gibbs; John Y Kuwada; Carsten G Bonnemann; Eva L Feldman
Journal:  PLoS Genet       Date:  2009-02-06       Impact factor: 5.917

10.  AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis.

Authors:  Anna Buj-Bello; Françoise Fougerousse; Yannick Schwab; Nadia Messaddeq; Danièle Spehner; Christopher R Pierson; Muriel Durand; Christine Kretz; Olivier Danos; Anne-Marie Douar; Alan H Beggs; Patrick Schultz; Marie Montus; Patrice Denèfle; Jean-Louis Mandel
Journal:  Hum Mol Genet       Date:  2008-04-22       Impact factor: 6.150
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  1 in total

1.  Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs.

Authors:  Martin K Childers; Alan H Beggs; Ana Buj-Bello
Journal:  Ann Transl Med       Date:  2015-10
  1 in total

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