Current Genome Editing Tools in Gene Therapy: New Approaches to Treat Cancer
Nickolai A Barlev.
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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