Genetic correction using engineered nucleases for gene therapy applications.
Dev Growth Differ
; 56(1): 63-77, 2014 Jan.
Article
em En
| MEDLINE
| ID: mdl-24329887
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.
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Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
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Engenharia Genética
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Endodesoxirribonucleases
Idioma:
En
Ano de publicação:
2014
Tipo de documento:
Article