Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid developme...

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Bibliographic Details
Published in:The Journal of clinical investigation 2014-10, Vol.124 (10), p.4154-4161
Main Authors: Gupta, Rajat M, Musunuru, Kiran
Format: Article
Language:English
Subjects:
Animals
Antibiotics
Bacterial Proteins - metabolism
Base Sequence
Biomedical research
CRISPR-Cas Systems
Deoxyribonucleases - metabolism
Deoxyribonucleic acid
DNA
DNA - chemistry
DNA Helicases - metabolism
DNA Repair
DNA-Binding Proteins - metabolism
Editing
Efficiency
Endonucleases - genetics
Forecasts and trends
Gene expression
Genetic engineering
Genetic Engineering - methods
Genome
Genomes
Humans
Innovations
Medical laboratory technology
Medical technology
Molecular Sequence Data
Mutation
Nucleases
Properties
Protein Binding
Protein Multimerization
Protein Structure, Tertiary
Science in Medicine
Sequence Homology, Nucleic Acid
Site selection
Stem cells
Transcription, Genetic
Zinc Fingers
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Summary:The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid development of the field has allowed for highly efficient, precise, and now cost-effective means by which to generate human and animal models of disease using these technologies. This review will outline the recent development of genome-editing technology, culminating with the use of CRISPR-Cas9 to generate novel mammalian models of disease. While the road to using this same technology for treatment of human disease is long, the pace of innovation over the past five years and early successes in model systems build anticipation for this prospect.
ISSN:0021-9738
1558-8238
DOI:10.1172/jci72992