Genetic correction using engineered nucleases for gene therapy applications. (11th December 2013)

• Record Type: Journal Article
• Authors: Lisa Li, Hongmei; Nakano, Takao; Hotta, Akitsu
• Is Part Of: Development growth and differentiation. Volume 56:Number 1(2014)
• Page Start: 63
• Page End: 77
• Publication Date: 2013-12-11
• Subjects: Embryology -- Periodicals; Developmental biology -- Periodicals; Growth -- Periodicals; 574.3
• DOI: 10.1111/dgd.12107
• Languages: English
• ISSNs: 0012-1592

Abstract

<abstract abstract-type="main" id="dgd12107-abs-0001"> <title> <x xml:space="preserve">Abstract</x> </title> <p>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.</p> </abstract>