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Adeno-associated Virus Vectors for Gene Transfer into Erythroid Cells

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy

Part of the book series: Current Topics in Microbiology and Immunology ((CT MICROBIOLOGY,volume 218))

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Abstract

Adeno-associated virus (AAV) vectors have the potential to treat a broad range of genetic diseases, among which, those directed toward blood cells currently hold the greatest promise. There are several reasons why the prospect of transducing blood cells merits such extensive interest. First, the target cells are easily accessible. Populations of cells can be removed from the patient, manipulated ex vivo, and subsequently returned to the patient. They can also be enriched for the cell types of interest, cultured, expanded, and transduced with a number of experimental gene therapy vectors. Perhaps more importantly, pluripotent stem cell populations have been identified which can divide and differentiate to repopulate the blood with the different lineages of cells which make up this complex system.

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Author information

Authors and Affiliations

  1. Gene-Therapy Center and Department of Pharmacology, University of North Carolina, 27599, Chapel Hill, NC, USA

    D. M. McCarty & R. J. Samulski

Authors
  1. D. M. McCarty
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  2. R. J. Samulski
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Editors and Affiliations

  1. Hearst Microbiology Research Center, Department of Microbiology, Cornell University Medical College, 1300 York Avenue, Box 62, 10021, New York, NY, USA

    Kenneth I. Berns M.D., Ph.D.  & Catherine Giraud Ph.D.  & 

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© 1996 Springer-Verlag Berlin Heidelberg

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McCarty, D.M., Samulski, R.J. (1996). Adeno-associated Virus Vectors for Gene Transfer into Erythroid Cells. In: Berns, K.I., Giraud, C. (eds) Adeno-Associated Virus (AAV) Vectors in Gene Therapy. Current Topics in Microbiology and Immunology, vol 218. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-80207-2_6

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  • DOI: https://doi.org/10.1007/978-3-642-80207-2_6

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-80209-6

  • Online ISBN: 978-3-642-80207-2

  • eBook Packages: Springer Book Archive

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