Advances in targeted genome editing

Pablo Perez-Pinera, David G. Ousterout, Charles A. Gersbach

Research output: Contribution to journalReview article

Abstract

New technologies have recently emerged that enable targeted editing of genomes in diverse systems. This includes precise manipulation of gene sequences in their natural chromosomal context and addition of transgenes to specific genomic loci. This progress has been facilitated by advances in engineering targeted nucleases with programmable, site-specific DNA-binding domains, including zinc finger proteins and transcription activator-like effectors (TALEs). Recent improvements have enhanced nuclease performance, accelerated nuclease assembly, and lowered the cost of genome editing. These advances are driving new approaches to many areas of biotechnology, including biopharmaceutical production, agriculture, creation of transgenic organisms and cell lines, and studies of genome structure, regulation, and function. Genome editing is also being investigated in preclinical and clinical gene therapies for many diseases.

Original languageEnglish (US)
Pages (from-to)268-277
Number of pages10
JournalCurrent Opinion in Chemical Biology
Volume16
Issue number3-4
DOIs
StatePublished - Aug 1 2012

ASJC Scopus subject areas

  • Analytical Chemistry
  • Biochemistry

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