• Methods Mol. Biol. · Jan 2008

    Design of trans-splicing adeno-associated viral vectors for Duchenne muscular dystrophy gene therapy.

    • Yi Lai, Dejia Li, Yongping Yue, and Dongsheng Duan.
    • Department of Molecular Microbiology and Immunology, University of Missouri, Columbia, MO, USA.
    • Methods Mol. Biol. 2008 Jan 1; 433: 259-75.

    AbstractThe development of trans-splicing vectors opens the door for delivering a large therapeutic gene with adeno-associated viral vectors (AAV). One potential application is to deliver the 6 kb mini-dystrophin gene for Duchenne muscular dystrophy (DMD) gene therapy. However, early attempts have been very disappointing because of low transduction efficiency. We have recently identified mRNA accumulation as a critical barrier for the trans-splicing AAV vectors. This barrier can be overcome by rational selection of the gene splitting site. Here we outline a detailed RNase protection assay-based strategy to determine the optimal gene splitting site for the mini-dystrophin gene. We also provide methods to evaluate transduction efficiency of the mini-dystrophin trans-splicing vectors in mdx mouse, a model for DMD.

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