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Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.

Thomas Gaj1, Benjamin E Epstein2, David V Schaffer1,2,3,4

  • 1Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|September 17, 2015
PubMed
Summary
This summary is machine-generated.

Adeno-associated virus (AAV) vectors efficiently stimulate homologous recombination for gene targeting. Combining AAV with targeted nucleases offers powerful genome engineering for next-generation gene therapy.

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Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Genomics

Background:

  • Adeno-associated virus (AAV) vectors are recognized for their therapeutic gene delivery capabilities.
  • AAV vectors possess a unique ability to stimulate high-efficiency homologous recombination in mammalian cells, a process termed AAV-mediated gene targeting.
  • This gene targeting has facilitated diverse genomic modifications both in vitro and in vivo.

Purpose of the Study:

  • To review the key properties of AAV vectors that make them uniquely useful for genome editing.
  • To highlight the wide range of genome engineering applications enabled by AAV technology.
  • To discuss the potential of combining AAV with targeted nucleases for advanced gene therapy.

Main Methods:

  • Review of existing literature on AAV vector properties and genome engineering applications.
  • Analysis of AAV's mechanism in stimulating homologous recombination.
  • Exploration of the synergy between AAV and targeted nuclease technologies.

Main Results:

  • AAV vectors exhibit inherent high efficiency in stimulating homologous recombination for gene targeting.
  • AAV-mediated gene targeting has been successfully applied for various genomic modifications in cellular and animal models.
  • The integration of targeted nucleases with AAV vectors presents a promising avenue for clinical gene therapy.

Conclusions:

  • AAV vectors possess unique characteristics that are highly advantageous for genome editing applications.
  • The combination of AAV vectors with targeted nucleases is a rapidly advancing field with significant potential for clinical translation in gene therapy.