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J Genet Genomics. 2012 Jun 20;39(6):269-74. doi: 10.1016/j.jgg.2012.05.004. Epub 2012 May 18.

Targeted genome editing by recombinant adeno-associated virus (rAAV) vectors for generating genetically modified pigs.

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1
Department of Biomedicine, Aarhus University, Wilhelm Meyers Alle 4, DK-8000 Aarhus C, Denmark. alun@hum-gen.au.dk

Abstract

Recombinant adeno-associated virus (rAAV) vectors have been extensively used for experimental gene therapy of inherited human diseases. Several advantages, such as simple vector construction, high targeting frequency by homologous recombination, and applicability to many cell types, make rAAV an attractive approach for targeted genome editing. Combined with cloning by somatic cell nuclear transfer (SCNT), this technology has recently been successfully adapted to generate gene-targeted pigs as models for cystic fibrosis, hereditary tyrosinemia type 1, and breast cancer. This review summarizes the development of rAAV for targeted genome editing in mammalian cells and provides strategies for enhancing the rAAV-mediated targeting frequency by homologous recombination. We discuss current development and application of the rAAV vectors for targeted genome editing in porcine primary fibroblasts, which are subsequently used as donor cells for SCNT to generate cloned genetically designed pigs and provide positive perspectives for the generation of gene-targeted pigs with rAAV in the future.

PMID:
22749014
DOI:
10.1016/j.jgg.2012.05.004
[Indexed for MEDLINE]
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