Literature DB >> 22297820

Signs of progress in gene therapy for muscular dystrophy also warrant caution.

Hansell H Stedman1, Barry J Byrne.   

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Year:  2012        PMID： 22297820      PMCID： PMC3277231          DOI： 10.1038/mt.2011.307

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  20 in total

1.  Dystrophin immunity in Duchenne's muscular dystrophy.

Authors:  Jerry R Mendell; Katherine Campbell; Louise Rodino-Klapac; Zarife Sahenk; Chris Shilling; Sarah Lewis; Dawn Bowles; Steven Gray; Chengwen Li; Gloria Galloway; Vinod Malik; Brian Coley; K Reed Clark; Juan Li; Xiao Xiao; Jade Samulski; Scott W McPhee; R Jude Samulski; Christopher M Walker
Journal:  N Engl J Med       Date:  2010-10-07       Impact factor: 91.245

2.  Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector.

Authors:  Leonard T Su; Kapil Gopal; Zhonglin Wang; Xiaoqing Yin; Anthony Nelson; Benjamin W Kozyak; James M Burkman; Marilyn A Mitchell; David W Low; Charles R Bridges; Hansell H Stedman
Journal:  Circulation       Date:  2005-09-12       Impact factor: 29.690

3.  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

4.  Mitigation of muscular dystrophy in mice by SERCA overexpression in skeletal muscle.

Authors:  Sanjeewa A Goonasekera; Chi K Lam; Douglas P Millay; Michelle A Sargent; Roger J Hajjar; Evangelia G Kranias; Jeffery D Molkentin
Journal:  J Clin Invest       Date:  2011-03       Impact factor: 14.808

5.  Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.

Authors:  Zheng Fan; Keith Kocis; Robert Valley; James F Howard; Manisha Chopra; Hongyu An; Weili Lin; Joseph Muenzer; William Powers
Journal:  Mol Ther       Date:  2011-07-19       Impact factor: 11.454

6.  Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs.

Authors:  G Acsadi; G Dickson; D R Love; A Jani; F S Walsh; A Gurusinghe; J A Wolff; K E Davies
Journal:  Nature       Date:  1991-08-29       Impact factor: 49.962

7.  Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.

Authors:  Valder R Arruda; Hansell H Stedman; Timothy C Nichols; Mark E Haskins; Matthew Nicholson; Roland W Herzog; Linda B Couto; Katherine A High
Journal:  Blood       Date:  2004-10-12       Impact factor: 22.113

8.  The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein.

Authors:  M Koenig; A P Monaco; L M Kunkel
Journal:  Cell       Date:  1988-04-22       Impact factor: 41.582

9.  Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene.

Authors:  A P Monaco; R L Neve; C Colletti-Feener; C J Bertelson; D M Kurnit; L M Kunkel
Journal:  Nature       Date:  1986 Oct 16-22       Impact factor: 49.962

10.  Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Authors:  Amit C Nathwani; Edward G D Tuddenham; Savita Rangarajan; Cecilia Rosales; Jenny McIntosh; David C Linch; Pratima Chowdary; Anne Riddell; Arnulfo Jaquilmac Pie; Chris Harrington; James O'Beirne; Keith Smith; John Pasi; Bertil Glader; Pradip Rustagi; Catherine Y C Ng; Mark A Kay; Junfang Zhou; Yunyu Spence; Christopher L Morton; James Allay; John Coleman; Susan Sleep; John M Cunningham; Deokumar Srivastava; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; John T Gray; Ulrike M Reiss; Arthur W Nienhuis; Andrew M Davidoff
Journal:  N Engl J Med       Date:  2011-12-10       Impact factor: 176.079
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  1 in total

Review 1.  Gene Therapy for Heart Failure: New Perspectives.

Authors:  Khatia Gabisonia; Fabio A Recchia
Journal:  Curr Heart Fail Rep       Date:  2018-12
  1 in total

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