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Biomacromolecules · 2019

Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery?

Biomacromolecules, 20(9): 3333-3339 PMID 31342740 DOI 10.1021/acs.biomac.9b00783

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Аннотация

Genome-editing technologies hold tremendous potential for treating genetic diseases. However, the efficient and safe delivery of genome-editing elements to the location of interest, and the achievement of specific targeted gene correction without off-target side effect remains a big challenge. In this Perspective, we highlight recent developments and discuss the challenges of nonviral nanoparticles for the delivery of genome-editing tools. Finally, we will propose promising strategies to improve the delivery efficacy and advance the clinical translation of gene-editing technology.

Цитирование