Current Genome Editing Tools in Gene Therapy: New Approaches to Treat Cancer

Author(s): Oleg Shuvalov, Alexey Petukhov, Alexandra Daks, Olga Fedorova, Alexander Ermakov, Gerry Melino and Nickolai A Barlev

Volume 15, Issue 5, 2015

Page: [511 - 529] Pages: 19

DOI: 10.2174/1566523215666150818110241

Price: $65


Gene therapy suggests a promising approach to treat genetic diseases by applying genes as pharmaceuticals. Cancer is a complex disease, which strongly depends on a particular genetic make-up and hence can be treated with gene therapy. From about 2,000 clinical trials carried out so far, more than 60% were cancer targeted. Development of precise and effective gene therapy approaches is intimately connected with achievements in the molecular biology techniques. The field of gene therapy was recently revolutionized by the introduction of “programmable” nucleases, including ZFNs, TALENs, and CRISPR, which target specific genomic loci with high efficacy and precision. Furthermore, when combined with DNA transposons for the delivery purposes into cells, these programmable nucleases represent a promising alternative to the conventional viral-mediated gene delivery. In addition to “programmable” nucleases, a new class of TALE- and CRISPR-based “artificial transcription effectors” has been developed to mediate precise regulation of specific genes. In sum, these new molecular tools may be used in a wide plethora of gene therapy strategies. This review highlights the current status of novel genome editing tools and discusses their suitability and perspectives in respect to cancer gene therapy studies.

Keywords: Cancer gene therapy, Genome editing, Artificial transcriptional effectors, Sleeping beauty, PiggyBac, Mega nucleases, ZFNs, Tale, CRISPR/Cas9.

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