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Literature DB >> 16629052

Gene therapy for Duchenne muscular dystrophy: AAV leads the way.

Abstract

Over the past decade, adeno-associated virus (AAV) has become an extremely promising vector for gene therapy of many genetic disorders. This review summarizes the specific challenges that must be overcome to apply AAV gene therapy to Duchenne muscular dystrophy. Many of these challenges have been met successfully in animal studies, but further work is needed to translate these results into an effective clinical treatment.

Entities: Disease Species

Mesh：

Substances：
Dystrophin

Year: 2005 PMID： 16629052

Source DB: PubMed Journal: Acta Myol ISSN： 1128-2460

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Cited

7 in total

1. Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy.

Authors: Ida Luisa Rotundo; Stefania Faraso; Elvira De Leonibus; Gerardo Nigro; Carmen Vitiello; Alessio Lancioni; Daniele Di Napoli; Sigismondo Castaldo; Vincenzo Russo; Fabio Russo; Giulio Piluso; Alberto Auricchio; Vincenzo Nigro
Journal: PLoS One Date: 2011-09-09 Impact factor: 3.240

2. β1D chain increases α7β1 integrin and laminin and protects against sarcolemmal damage in mdx mice.

Authors: Jianming Liu; Derek J Milner; Marni D Boppart; Robert S Ross; Stephen J Kaufman
Journal: Hum Mol Genet Date: 2011-12-16 Impact factor: 6.150

3. Inhibition of antigen presentation during AAV gene therapy using virus peptides.

Authors: Wenwei Shao; Xiaojing Chen; Richard J Samulski; Matthew L Hirsch; Chengwen Li
Journal: Hum Mol Genet Date: 2018-02-15 Impact factor: 6.150

4. Deletion of Galgt2 (B4Galnt2) reduces muscle growth in response to acute injury and increases muscle inflammation and pathology in dystrophin-deficient mice.

Authors: Rui Xu; Neha Singhal; Yelda Serinagaoglu; Kumaran Chandrasekharan; Mandar Joshi; John A Bauer; Paulus M L Janssen; Paul T Martin
Journal: Am J Pathol Date: 2015-10 Impact factor: 4.307

Gene therapy for Duchenne muscular dystrophy: AAV leads the way.

1. Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy.

2. β1D chain increases α7β1 integrin and laminin and protects against sarcolemmal damage in mdx mice.

3. Inhibition of antigen presentation during AAV gene therapy using virus peptides.

4. Deletion of Galgt2 (B4Galnt2) reduces muscle growth in response to acute injury and increases muscle inflammation and pathology in dystrophin-deficient mice.

5. Manipulating gene expression in projection-specific neuronal populations using combinatorial viral approaches.

6. The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins.

Review 7. Progress in therapeutic antisense applications for neuromuscular disorders.