Review

Gene Therapy (2009) 16, 581–588; doi:10.1038/gt.2009.16; published online 19 February 2009

Progress and prospects: techniques for site-directed mutagenesis in animal models

Z Yan1,2, X Sun1,2 and J F Engelhardt1,2

  1. 1Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA, USA
  2. 2Center for Gene Therapy, Carver College of Medicine, University of Iowa, Iowa City, IA, USA

Correspondence: Professor JF Engelhardt, Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Bowen Science Building, 51 Newton Road, Iowa City, IA 52242-1109, USA. E-mail: john-engelhardt@uiowa.edu

Received 17 November 2008; Revised 22 January 2009; Accepted 26 January 2009; Published online 19 February 2009.

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Abstract

In the past 2 years, new gene-targeting approaches using adeno-associated virus and designer zinc-finger nucleases have been successfully applied to the production of genetically modified ferrets, pigs, mice and zebrafish. Gene targeting using these tools has been combined with somatic cell nuclear transfer and germ cell transplantation to generate gene-targeted animal models. These new technical advances, which do not require the generation of embryonic stem cell-derived chimeras, will greatly accelerate the production of non-mouse animal models for biomedical research.

Keywords:

gene targeting, recombinant adeno-associated virus, animal models, zinc-finger nucleases, homologous recombination, somatic cell nuclear transfer

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