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Gene editing technology for improving life quality: A dream coming true?
Author(s) -
Ramezankhani Roya,
Minaei Neda,
Haddadi Mahnaz,
Torabi Shukoofeh,
Hesaraki Mahdi,
Mirzaei Hamed,
Vosough Massoud,
Verfaillie Catherine M.
Publication year - 2021
Publication title -
clinical genetics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.543
H-Index - 102
eISSN - 1399-0004
pISSN - 0009-9163
DOI - 10.1111/cge.13794
Subject(s) - transcription activator like effector nuclease , genome editing , crispr , zinc finger nuclease , computational biology , cas9 , effector , biology , computer science , genetics , gene , bioinformatics , immunology
The fact that monogenic diseases are related to mutations in one specific gene, make gene correction one of the promising strategies in the future to treat genetic diseases or alleviate their symptoms. From this perspective, and along with recent advances in technology, genome editing tools have gained momentum and developed fast. In fact, clustered regularly interspaced short palindromic repeats‐associated protein 9 (CRISPR/Cas9), transcription activator‐like effector nucleases (TALENs), and zinc‐finger nucleases (ZFNs) are regarded as novel technologies which are able to correct a number of genetic aberrations in vitro and in vivo. The number of ongoing clinical trials employing these tools has been increased showing the encouraging outcomes of these tools. However, there are still some major challenges with respect to the safety profile and directed delivery of them. In this paper, we provided updated information regarding the history, nature, methods of delivery, and application of the above‐mentioned gene editing tools along with the meganucleases (an older similar tool) based on published in vitro and in vivo studies and introduced clinical trials which employed these technologies.