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Genetic correction using engineered nucleases for gene therapy applications
Author(s) -
Lisa Li Hongmei,
Nakano Takao,
Hotta Akitsu
Publication year - 2014
Publication title -
development, growth and differentiation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.864
H-Index - 66
eISSN - 1440-169X
pISSN - 0012-1592
DOI - 10.1111/dgd.12107
Subject(s) - transcription activator like effector nuclease , zinc finger nuclease , genome editing , crispr , genetic enhancement , cas9 , biology , computational biology , genome engineering , gene , genetics , genetic engineering , genome , human genetics
Genetic mutations in humans are associated with congenital disorders and phenotypic traits. Gene therapy holds the promise to cure such genetic disorders, although it has suffered from several technical limitations for decades. Recent progress in gene editing technology using tailor‐made nucleases, such as meganucleases ( MN s), zinc finger nucleases ( ZFN s), TAL effector nucleases ( TALEN s) and, more recently, CRISPR /Cas9, has significantly broadened our ability to precisely modify target sites in the human genome. In this review, we summarize recent progress in gene correction approaches of the human genome, with a particular emphasis on the clinical applications of gene therapy.