Genome engineering using Adeno-associated virus: Basic and clinical research applications

Thomas Gaj, Benjamin E. Epstein, David V. Schaffer

Research output: Contribution to journalReview articlepeer-review


In addition to their broad potential for therapeutic gene delivery, adeno-associated virus (AAV) vectors possess the innate ability to stimulate homologous recombination in mammalian cells at high efficiencies. This process - referred to as AAV-mediated gene targeting - has enabled the introduction of a diverse array of genomic modifications both in vitro and in vivo. With the recent emergence of targeted nucleases, AAV-mediated genome engineering is poised for clinical translation. Here, we review key properties of AAV vectors that underscore its unique utility in genome editing. We highlight the broad range of genome engineering applications facilitated by this technology and discuss the strong potential for unifying AAV with targeted nucleases for next-generation gene therapy.

Original languageEnglish (US)
Pages (from-to)458-464
Number of pages7
JournalMolecular Therapy
Issue number3
StatePublished - Mar 1 2016
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery


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