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Non-Viral Delivery To Enable Genome Editing

Author:
Rui, Yuan, Wilson, David R., Green, Jordan J.
Source:
Trends in biotechnology 2019 v.37 no.3 pp. 281-293
ISSN:
0167-7799
Subject:
DNA-binding proteins, biotechnology, cell nucleus, gene editing, gene expression, genes, nucleases, patient care, translation (genetics)
Abstract:
Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.
Agid:
6160202