In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing
Why the study?
Does in vivo base editing of the Pcsk9 gene reduce plasma PCSK9 and cholesterol levels in adult mice?
Population
Adult mice and cultured cells (including human-induced pluripotent stem cells)
Design
Preclinical
Authors
Discussion
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Member takes
Implication
Provides proof-of-concept for a potentially safer alternative to traditional CRISPR-Cas9.
Structured PICO
Does in vivo base editing of the Pcsk9 gene reduce plasma PCSK9 and cholesterol levels in adult mice?
P
Population
Adult mice and cultured cells (including human-induced pluripotent stem cells)
I
Intervention
In vivo base editing (CRISPR-Cas9 fused to a cytosine deaminase domain) targeting the Pcsk9 gene delivered into the liver
O
Outcome
Plasma PCSK9 protein levels and plasma cholesterol levelssurrogate
In vivo base editing of the Pcsk9 gene effectively reduces PCSK9 and cholesterol levels in mice without off-target mutations, demonstrating a potentially safer alternative to traditional CRISPR-Cas9 genome editing.
Cite This Study
Chadwick et al. (2017) studied this question.