Cancer gene therapy: innovations in therapeutic delivery of CRISPR-Cas9

Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent development of a genome editing system using clustered regularly interspersed palindromic repeats (CRISPR) has made gene therapy for human diseases m...

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Bibliographic Details
Published inDrug discovery today. Disease models Vol. 21; pp. 17 - 21
Main Authors Lindsay-Mosher, Nicole, Su, Cathy
Format Journal Article
LanguageEnglish
Published Elsevier Ltd 2016
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Summary:Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent development of a genome editing system using clustered regularly interspersed palindromic repeats (CRISPR) has made gene therapy for human diseases much more feasible. To realize the full potential of CRISPR and CRISPR-associated systems (Cas) for cancer treatment, however, this gene editing system must be efficiently and safely delivered to cancer cells in vivo. Here we review innovations made in the design of both viral and non-viral vectors to accommodate CRISPR-Cas systems, addressing the challenges of size constraints, immunogenicity, and specificity.
ISSN:1740-6757
1740-6757
DOI:10.1016/j.ddmod.2017.02.009