基因治疗目前的基因组编辑工具：治疗癌症的新方法

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

Volume: 15 Issue: 5

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

Affiliation:

关键词: 肿瘤的基因治疗，基因编辑，人工转录因子，“睡美人”转座子，PiggyBac，大型核酸酶，ZFNs，Tale，CRISPR/Cas9。

摘要: 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.

Cite this article as:

Shuvalov Oleg, Petukhov Alexey, Daks Alexandra, Fedorova Olga, Ermakov Alexander and Melino Gerry, Current Genome Editing Tools in Gene Therapy: New Approaches to Treat Cancer, Current Gene Therapy 2015; 15 (5) . https://dx.doi.org/10.2174/1566523215666150818110241