CRISPR genome engineering and viral gene delivery: a case of mutual attraction

The adaptation of the CRISPR/Cas9 DNA engineering machinery for mammalian cells has revolutionized our approaches to low- or high-throughput genome annotation and paved the way for conceptually novel therapeutic strategies. A large part of the attraction of CRISPR stems from the small size of its tw...

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Bibliographic Details
Main Authors: Schmidt, Florian (Author) , Grimm, Dirk (Author)
Format: Article (Journal)
Language:English
Published: 6 February 2015
In: Biotechnology journal
Year: 2015, Volume: 10, Issue: 2, Pages: 258-272
ISSN:1860-7314
DOI:10.1002/biot.201400529
Online Access:Volltext
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Author Notes:Florian Schmidt, Dirk Grimm
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Summary:The adaptation of the CRISPR/Cas9 DNA engineering machinery for mammalian cells has revolutionized our approaches to low- or high-throughput genome annotation and paved the way for conceptually novel therapeutic strategies. A large part of the attraction of CRISPR stems from the small size of its two core components - Cas9 and gRNA - and hence its compatibility with virtually any available viral vector delivery system. As a result, over the past two years, four major classes of viral vectors have already been engineered and applied as CRISPR delivery tools - retroviruses, lentiviruses, adenoviruses, and adeno-associated viruses (AAVs). The juxtaposition of these two technologies reflects a case of tremendous mutual attraction and holds unprecedented promises for biology and medicine. Here, we provide an overview of the state-of-the-art of this rapidly emerging field, from a comparative description of the principal vector designs, to a synopsis of some of the most exciting applications that were reported to date, including the use of viral CRISPR vectors for genome-wide loss-of-function screens, multiplexed gene editing or disease modeling in animals. Once specificity and safety have been improved further, viral vector-mediated in vitro/in vivo CRISPR delivery and expression promise to radically transform basic and applied biomedical research.
Item Description:Gesehen am 03.07.2017
Physical Description:Online Resource
ISSN:1860-7314
DOI:10.1002/biot.201400529