One-shot, one cure with genome editing for dyslipidemia
- PMID: 25516627
- DOI: 10.1161/CIRCGENETICS.114.000958
One-shot, one cure with genome editing for dyslipidemia
Keywords: PCSK9 protein, human; gene targeting; gene, suppressor; genetic therapy; lipids.
Comment on
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Mutations in PCSK9 cause autosomal dominant hypercholesterolemia.Nat Genet. 2003 Jun;34(2):154-6. doi: 10.1038/ng1161. Nat Genet. 2003. PMID: 12730697
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Low LDL cholesterol in individuals of African descent resulting from frequent nonsense mutations in PCSK9.Nat Genet. 2005 Feb;37(2):161-5. doi: 10.1038/ng1509. Epub 2005 Jan 16. Nat Genet. 2005. PMID: 15654334
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Reduction of low-density lipoprotein cholesterol by monoclonal antibody inhibition of PCSK9.Annu Rev Med. 2014;65:417-31. doi: 10.1146/annurev-med-022613-090402. Annu Rev Med. 2014. PMID: 24422577 Review.
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Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing.Circ Res. 2014 Aug 15;115(5):488-92. doi: 10.1161/CIRCRESAHA.115.304351. Epub 2014 Jun 10. Circ Res. 2014. PMID: 24916110 Free PMC article.
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