Abstract
CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats, CRISPR/CRISPR-associated nuclease 9), a kind of acquired immune system peculiar to bacteria, has been improved as a new genetic engineering tool. It is known as the scissor of the god because of its huge advantage of simple design, convenient operation, low cost and high efficiency. CRISPR/Cas9 technology has already been widely promoted and applied in gene function research, animal modeling, gene therapy, and other fields. This paper highlights the origin, development and mechanism of CRISPR/Cas9 genome editing technology, and summarizes the latest application in gene therapy for human diseases, especially for the childhood-onset monogenetic diseases, so as to provide a reference for its wider research.(Chin J Lab Med, 2016, 39: 243-245) Key words: CRISPR/Cas9; Gene editing; Monogenetic disease
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