Literature DB >> 20543898

Could gene therapy be the future for muscular dystrophy?

Amanda M Haidet1, Jerry R Mendell, Brian K Kaspar.   

Abstract

Entities:  

Year:  2010        PMID： 20543898      PMCID： PMC2883255          DOI： 10.2217/thy.10.14

Source DB:  PubMed          Journal:  Therapy        ISSN： 1475-0708


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  31 in total

Review 1.  Dystrophin-glycoprotein complex: post-translational processing and dystroglycan function.

Authors:  Daniel E Michele; Kevin P Campbell
Journal:  J Biol Chem       Date:  2003-01-29       Impact factor: 5.157

2.  Regulation of muscle growth by multiple ligands signaling through activin type II receptors.

Authors:  Se-Jin Lee; Lori A Reed; Monique V Davies; Stefan Girgenrath; Mary E P Goad; Kathy N Tomkinson; Jill F Wright; Christopher Barker; Gregory Ehrmantraut; James Holmstrom; Betty Trowell; Barry Gertz; Man-Shiow Jiang; Suzanne M Sebald; Martin Matzuk; En Li; Li-Fang Liang; Edwin Quattlebaum; Ronald L Stotish; Neil M Wolfman
Journal:  Proc Natl Acad Sci U S A       Date:  2005-12-05       Impact factor: 11.205

3.  Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo.

Authors:  Christina A Pacak; Cathryn S Mah; Bijoy D Thattaliyath; Thomas J Conlon; Melissa A Lewis; Denise E Cloutier; Irene Zolotukhin; Alice F Tarantal; Barry J Byrne
Journal:  Circ Res       Date:  2006-07-27       Impact factor: 17.367

4.  Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice.

Authors:  Ciaran D Scallan; Haiyan Jiang; Tongyao Liu; Susannah Patarroyo-White; Jurg M Sommer; Shangzhen Zhou; Linda B Couto; Glenn F Pierce
Journal:  Blood       Date:  2005-10-25       Impact factor: 22.113

5.  Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.

Authors:  Haiyan Jiang; Linda B Couto; Susannah Patarroyo-White; Tongyao Liu; Dea Nagy; Joseph A Vargas; Shangzhen Zhou; Ciaran D Scallan; Jurg Sommer; Sharmila Vijay; Federico Mingozzi; Katherine A High; Glenn F Pierce
Journal:  Blood       Date:  2006-07-25       Impact factor: 22.113

6.  Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.

Authors:  Scott Q Harper; Michael A Hauser; Christiana DelloRusso; Dongsheng Duan; Robert W Crawford; Stephanie F Phelps; Hollie A Harper; Ann S Robinson; John F Engelhardt; Susan V Brooks; Jeffrey S Chamberlain
Journal:  Nat Med       Date:  2002-03       Impact factor: 53.440

7.  Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals.

Authors:  M Koenig; E P Hoffman; C J Bertelson; A P Monaco; C Feener; L M Kunkel
Journal:  Cell       Date:  1987-07-31       Impact factor: 41.582

8.  Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression.

Authors:  En Kimura; Sheng Li; Paul Gregorevic; Brent M Fall; Jeffrey S Chamberlain
Journal:  Mol Ther       Date:  2009-11-03       Impact factor: 11.454

9.  Loss of myostatin attenuates severity of muscular dystrophy in mdx mice.

Authors:  Kathryn R Wagner; Alexandra C McPherron; Nicole Winik; Se-Jin Lee
Journal:  Ann Neurol       Date:  2002-12       Impact factor: 10.422

10.  Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice.

Authors:  S F Phelps; M A Hauser; N M Cole; J A Rafael; R T Hinkle; J A Faulkner; J S Chamberlain
Journal:  Hum Mol Genet       Date:  1995-08       Impact factor: 6.150
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  1 in total

1.  Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions.

Authors:  Lei Xu; Pei Juan Lu; Chi-Hsien Wang; Elizabeth Keramaris; Chunping Qiao; Bin Xiao; Derek J Blake; Xiao Xiao; Qi Long Lu
Journal:  Mol Ther       Date:  2013-07-02       Impact factor: 11.454
  1 in total

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