A new world order: Tailored gene targeting and regulation using CRISPR (Editorial)

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Author(s)
Weinberg, MS
Morris, KV
Griffith University Author(s)
Year published
2014
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Not since the discovery of RNA interference has a new technology made such a powerful impact in the molecular biosciences. The RNA-guided, modified type II prokaryotic clustered regularly interspaced palindromic repeats system (CRISPR) represents not only an exceptional tool but also potentially an important new therapeutic modality.
CRISPR, in its native function, provides adaptive immunity in bacteria by introducing targeted DNA mutations in pathogenic viruses and plasmids (reviewed in ref. 1). The breakthrough technology came in 2012 with the development of modified CRISPR components that worked in human cells. This ...
View more >Not since the discovery of RNA interference has a new technology made such a powerful impact in the molecular biosciences. The RNA-guided, modified type II prokaryotic clustered regularly interspaced palindromic repeats system (CRISPR) represents not only an exceptional tool but also potentially an important new therapeutic modality. CRISPR, in its native function, provides adaptive immunity in bacteria by introducing targeted DNA mutations in pathogenic viruses and plasmids (reviewed in ref. 1). The breakthrough technology came in 2012 with the development of modified CRISPR components that worked in human cells. This simplified system was found to be easily adapted into expression vectors to target any DNA sequence from virtually any organism, thereby greatly expanding its function and utility. To date this system has been used in human cells to target gene excision, activating and repressing proteins, as well as to instill gene fusions.
View less >
View more >Not since the discovery of RNA interference has a new technology made such a powerful impact in the molecular biosciences. The RNA-guided, modified type II prokaryotic clustered regularly interspaced palindromic repeats system (CRISPR) represents not only an exceptional tool but also potentially an important new therapeutic modality. CRISPR, in its native function, provides adaptive immunity in bacteria by introducing targeted DNA mutations in pathogenic viruses and plasmids (reviewed in ref. 1). The breakthrough technology came in 2012 with the development of modified CRISPR components that worked in human cells. This simplified system was found to be easily adapted into expression vectors to target any DNA sequence from virtually any organism, thereby greatly expanding its function and utility. To date this system has been used in human cells to target gene excision, activating and repressing proteins, as well as to instill gene fusions.
View less >
Journal Title
Molecular Therapy
Volume
22
Issue
5
Copyright Statement
© 2014, The American Society of Gene & Cell Therapy. Published by Elsevier Inc. All rights reserved. Licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International Licence, which permits unrestricted, non-commercial use, distribution and reproduction in any medium, providing that the work is properly cited.
Subject
Biological sciences
Biomedical and clinical sciences