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Genome-Editing Technologies: Principles and Applications.

ABSTRACT: Targeted nucleases have provided researchers with the ability to manipulate virtually any genomic sequence, enabling the facile creation of isogenic cell lines and animal models for the study of human disease, and promoting exciting new possibilities for human gene therapy. Here we review three foundational technologies-clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), and zinc-finger nucleases (ZFNs). We discuss the engineering advances that facilitated their development and highlight several achievements in genome engineering that were made possible by these tools. We also consider artificial transcription factors, illustrating how this technology can complement targeted nucleases for synthetic biology and gene therapy.

SUBMITTER: Gaj T

PROVIDER: S-EPMC5131771 | biostudies-literature | 2016 Dec

REPOSITORIES: biostudies-literature

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Genome-Editing Technologies: Principles and Applications.

Gaj Thomas T Sirk Shannon J SJ Shui Sai-Lan SL Liu Jia J

Cold Spring Harbor perspectives in biology 20161201 12

Targeted nucleases have provided researchers with the ability to manipulate virtually any genomic sequence, enabling the facile creation of isogenic cell lines and animal models for the study of human disease, and promoting exciting new possibilities for human gene therapy. Here we review three foundational technologies-clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), and zinc-finger n ...[more]

PMID: 27908936

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