Current Genome Editing Tools in Gene Therapy: New Approaches to Treat Cancer
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: Artificial transcriptional effectors; CRISPR/Cas9; Cancer gene therapy; Genome editing; Mega nucleases; PiggyBac; Sleeping beauty; Tale; ZFNs
Document Type: Research Article
Publication date: 01 October 2015
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