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In Vivo Base Editing of PCSK9 as a Therapeutic Alternative to Genome Editing

OBJECTIVE: High-efficiency genome editing to disrupt therapeutic target genes such as PCSK9 has been demonstrated in preclinical animal models, but there are safety concerns due to the unpredictable nature of cellular repair of double-strand breaks, as well as off-target mutagenesis. Moreover, preci...

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Bibliografske podrobnosti
izdano v:Arterioscler Thromb Vasc Biol
Main Authors: Chadwick, Alexandra C., Wang, Xiao, Musunuru, Kiran
Format: Artigo
Jezik:Inglês
Izdano: 2017
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC5570639/
https://ncbi.nlm.nih.gov/pubmed/28751571
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1161/ATVBAHA.117.309881
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