title: Genetic correction using engineered nucleases for gene therapy applications.
abstract: 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 (MNs), zinc finger nucleases (ZFNs), TAL effector nucleases (TALENs) 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.
authors: Department of Reprogramming Science, Center for iPS cell Research and Applications (CiRA), Kyoto University, Kyoto, Japan; Japan Society for the Promotion of Science (JSPS), Tokyo, Japan.
authors: Hongmei Lisa
authors: HL
authors: Li
authors: Takao
authors: T
authors: Nakano
authors: Akitsu
authors: A
authors: Hotta
mesh_terms: D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics; D004706:Endodeoxyribonucleases / Q000737:chemistry / Q000235:genetics* / Q000378:metabolism*; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016335:Zinc Fingers
chemical_list: D004706:Endodeoxyribonucleases
keywords: gene repair; genome editing; human genetics