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Delivery Systems for Gene Therapy: Adeno‐Associated Virus

Gabriele Kroner‐Lux, Christopher Walsh, Richard Jude Samulski

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Abstract

The purpose of this chapter is to provide an overview of recombinant adeno-associated virus (rAAV) vectors and the current “state of the art” of their potential utility in gene therapy of hematopoietic disorders. The authors briefly review AAV biology and genetics and suggest more general reviews about AAV. The basic requirements that need to be fulfilled for gene therapy as treatment of hematopoietic disorders are presented.

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What this paper is about

The purpose of this chapter is to provide an overview of recombinant adeno-associated virus (rAAV) vectors and the current “state of the art” of their potential utility in gene therapy of hematopoietic disorders. The authors briefly review AAV biology and genetics and suggest more general reviews about AAV. The basic requirements that need to be fulfilled for gene therapy as treatment of hematopoietic disorders are presented.

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Available abstract

The purpose of this chapter is to provide an overview of recombinant adeno-associated virus (rAAV) vectors and the current “state of the art” of their potential utility in gene therapy of hematopoietic disorders. The authors briefly review AAV biology and genetics and suggest more general reviews about AAV. The basic requirements that need to be fulfilled for gene therapy as treatment of hematopoietic disorders are presented.

Key concepts: Adeno-associated virus, Genetic enhancement, Gene delivery, Virology, Haematopoiesis, Virus, Biology, Medicine

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