Therapeutic application of genome editing in dyslipidemia.

Autor: Whittaker MN; Department of Bioengineering, School of Engineering and Applied Science., Musunuru K; Cardiovascular Institute, Department of Medicine, and Department of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Jazyk: angličtina
Zdroj: Current opinion in lipidology [Curr Opin Lipidol] 2022 Apr 01; Vol. 33 (2), pp. 133-138.
DOI: 10.1097/MOL.0000000000000805
Abstrakt: Purpose of Review: To summarize recent advances with respect to the use of genome editing to modify blood lipid levels in vivo.
Recent Findings: Genome-editing technologies have been successfully used to target the PCSK9 gene in the livers of nonhuman primates and significantly reduce blood LDL cholesterol levels.
Summary: Multiple proof-of-concept nonhuman primate studies raise the prospect of genome editing empowering 'one-and-done' therapies for the treatment of dyslipidemic patients.
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Databáze: MEDLINE