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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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Veröffentlicht in:Arterioscler Thromb Vasc Biol
Hauptverfasser: Chadwick, Alexandra C., Wang, Xiao, Musunuru, Kiran
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
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Online Zugang: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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