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In Utero Muscle Gene Transfer

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Muscle Gene Therapy

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Abstract

Efficient, widespread transgene expression in the muscle is one of the major challenges of gene transfer for the treatment of genetic muscle disorders. A good example of this disease is the Duchenne muscular dystrophy (DMD), a progressive, degenerative disease whose clinical symptoms manifest after birth, but whose genetic defect causing the disease is present at conception. The availability of prenatal testing for genetic muscle diseases provides the basis for treatments in utero. In utero gene transfer has the potential to achieve widespread transgene expression in the muscle by accomplishing gene delivery when the tissue mass is small and the immune system is still immature. In this chapter, we present preclinical experience with gene delivery strategies to treat muscle disorders in utero. Important issues include experience with different gene delivery vectors in preclinical models, gene expression in muscle tissue, and effects on immunity.

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Acknowledgment

PRC is supported by VA resources (Pittsburgh VA Healthcare System, Pittsburgh, PA).

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  1. Department of Neurology, University of Pittsburgh, S-520 Biomedical Science Tower, Pittsburgh, PA, 15213, USA

    Bhanu Munil Koppanati

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  1. Bhanu Munil Koppanati
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  2. Paula R. Clemens
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Correspondence to Bhanu Munil Koppanati .

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  1. Molecular Microbiology & Immunology, University of Missouri, Hospital Dr. 1, Columbia, 65212, U.S.A.

    Dongsheng Duan

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Koppanati, B.M., Clemens, P.R. (2010). In Utero Muscle Gene Transfer. In: Duan, D. (eds) Muscle Gene Therapy. Springer, New York, NY. https://doi.org/10.1007/978-1-4419-1207-7_2

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