Gene Delivery Technologies for Efficient Genome Editing: Applications in Gene Therapy
Specific nucleases (SNs), including ZFNs, TALENs, and CRISPR (clustered regularly interspaced palindromic repeats), are powerful tools for genome editing (GE). These tools have achieved efficient gene repair and gene disruption of human primary cells. However, their efficiency and safety must be imp...
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| Main Authors: | , , , |
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| Format: | Ebooks |
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IntechOpen,
2016-05-18.
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| Summary: | Specific nucleases (SNs), including ZFNs, TALENs, and CRISPR (clustered regularly interspaced palindromic repeats), are powerful tools for genome editing (GE). These tools have achieved efficient gene repair and gene disruption of human primary cells. However, their efficiency and safety must be improved before translation into clinic. In particular, one of the main hurdles of GE technology is the delivery of the different components into the nucleus of target cells. Successful gene editing must be able to deliver the SNs and/or the donor DNA into a large number of target cells in order to have a therapeutic benefit. In addition, the delivery must be nontoxic and the SNs must be innocuous to the target cells. In this chapter, we will summarize the different ways to deliver SNs and donor DNA. |
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| Item Description: | https://mts.intechopen.com/articles/show/title/gene-delivery-technologies-for-efficient-genome-editing-applications-in-gene-therapy |