Literature DB >> 17234143

From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy.

Dongsheng Duan1.   

Abstract

The gene encoding dystrophin, which is altered in Duchenne muscular dystrophy, is one of the largest in mammalian species. Adeno-associated virus (AAV) is the smallest viral gene delivery vector with a "cargo" capacity of 6 kb and is the most effective gene delivery vehicle to muscle cells. Overlapping and trans-splicing AAV vector approaches are devised to do the "impossible."

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Mesh：

Year:  2006        PMID： 17234143      PMCID： PMC2581715     

Source DB:  PubMed          Journal:  Discov Med        ISSN： 1539-6509            Impact factor:   2.970


  7 in total

1.  ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES.

Authors:  R W ATCHISON; B C CASTO; W M HAMMON
Journal:  Science       Date:  1965-08-13       Impact factor: 47.728

2.  2004 William Allan Award address. Cloning of the DMD gene.

Authors:  Louis M Kunkel
Journal:  Am J Hum Genet       Date:  2005-02       Impact factor: 11.025

3.  rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

Authors:  Paul Gregorevic; James M Allen; Elina Minami; Michael J Blankinship; Miki Haraguchi; Leonard Meuse; Eric Finn; Marvin E Adams; Stanley C Froehner; Charles E Murry; Jeffrey S Chamberlain
Journal:  Nat Med       Date:  2006-07-02       Impact factor: 53.440

4.  Very mild muscular dystrophy associated with the deletion of 46% of dystrophin.

Authors:  S B England; L V Nicholson; M A Johnson; S M Forrest; D R Love; E E Zubrzycka-Gaarn; D E Bulman; J B Harris; K E Davies
Journal:  Nature       Date:  1990-01-11       Impact factor: 49.962

5.  C-terminal-truncated microdystrophin recruits dystrobrevin and syntrophin to the dystrophin-associated glycoprotein complex and reduces muscular dystrophy in symptomatic utrophin/dystrophin double-knockout mice.

Authors:  Yongping Yue; Mingju Liu; Dongsheng Duan
Journal:  Mol Ther       Date:  2006-03-23       Impact factor: 11.454

6.  Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

Authors:  Yi Lai; Yongping Yue; Mingju Liu; Arkasubhra Ghosh; John F Engelhardt; Jeffrey S Chamberlain; Dongsheng Duan
Journal:  Nat Biotechnol       Date:  2005-10-09       Impact factor: 54.908

7.  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 in total
  13 in total

1.  Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma.

Authors:  Yadong Zhang; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2011-10-24       Impact factor: 5.695

Review 2.  Progress in gene therapy of dystrophic heart disease.

Authors:  Y Lai; D Duan
Journal:  Gene Ther       Date:  2012-02-09       Impact factor: 5.250

3.  A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs.

Authors:  Jin-Hong Shin; Yongping Yue; Arun Srivastava; Bruce Smith; Yi Lai; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2011-12-14       Impact factor: 5.695

4.  Evaluation of microsatellite variation in the 1000 Genomes Project pilot studies is indicative of the quality and utility of the raw data and alignments.

Authors:  L J McIver; J W Fondon; M A Skinner; H R Garner
Journal:  Genomics       Date:  2011-01-09       Impact factor: 5.736

5.  Duchenne muscular dystrophy gene therapy: Lost in translation?

Authors:  Dongsheng Duan
Journal:  Res Rep Biol       Date:  2011-03

6.  Truncated dystrophins reduce muscle stiffness in the extensor digitorum longus muscle of mdx mice.

Authors:  Chady H Hakim; Dongsheng Duan
Journal:  J Appl Physiol (1985)       Date:  2012-12-06

7.  Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice.

Authors:  Brian Bostick; Yongping Yue; Yi Lai; Chun Long; Dejia Li; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2008-08       Impact factor: 5.695

8.  Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy.

Authors:  Arkasubhra Ghosh; Yongping Yue; Jin-Hong Shin; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2009-11       Impact factor: 5.695

9.  Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.

Authors:  Yongping Yue; Ibrahim M Binalsheikh; Stacey B Leach; Timothy L Domeier; Dongsheng Duan
Journal:  Expert Opin Orphan Drugs       Date:  2015-12-17       Impact factor: 0.694

10.  Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview.

Authors:  Dongsheng Duan
Journal:  Hum Gene Ther Clin Dev       Date:  2016-03       Impact factor: 5.032
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