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  • Cell-Based Therapy
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Cell-Based Therapy

Long-term phenotypic correction of rodent hemiparkinsonism by gene therapy using genetically modified myoblasts

Gene Therapy volume 7, pages 445–449 (2000)Cite this article

Abstract

Rat myoblasts were genetically modified to express tyrosine hydroxylase (TH) and produce dopamine in culture. Implanting TH gene-transfected myoblasts into the denervated striatum of 6-OHDA-lesioned rats significantly decreased rotational asymmetry by 50 to approximately 60%. Improvement persisted for up to 13 months. Genetically modified cells could survive and express transgene in the striatum as demonstrated by RT-PCR and immunohistochemical stain-ing. The dopamine content in the striatum tissue of the gene therapy group recovered to 49% of the normal level and was 25-fold higher than that of a control group receiving parental cells. Neither tumor formation nor immunorejection was observed in this study. These results show that myoblasts may be useful as gene carriers for ex vivo gene therapy in the CNS.

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Acknowledgements

We are very grateful to Dr K O'Malley (Washington University School of Medicine) for providing us with rat TH cDNA. We are also grateful to Lanying Sun, (Shanghai Institute of Biochemistry, Chinese Academy of Sciences) and Changfu Zhou (Shanghai Institute of Physiology, Chinese Academy of Sciences) for technical assistance and helpful advice. This work was supported by ‘86-3’ project (102-16-5 and BH-03-04-01) and the National Natural Science Foundation of China (No. 39230140).

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  1. L Cao

    Present address: CNS Gene Therapy Center, Thomas Jefferson University, USA

Authors and Affiliations

  1. Shanghai Institute of Biochemistry, Chinese Academy of Sciences, Shanghai, P.R. China

    L Cao, D-H Xu, X-Y Liu & Z-C Zheng

  2. Ruijin Hospital, Shanghai, P.R. China

    Y-C Zhao, Z-G Liu & S-D Chen

  3. Shanghai Institute of Physiology, Chinese Academy of Sciences, Shanghai, P.R. China

    Z-H Jiang

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Cao, L., Zhao, YC., Jiang, ZH. et al. Long-term phenotypic correction of rodent hemiparkinsonism by gene therapy using genetically modified myoblasts. Gene Ther 7, 445–449 (2000). https://doi.org/10.1038/sj.gt.3301096

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