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Review
. 2023 Oct 16;12(20):2466.
doi: 10.3390/cells12202466.

Development of New Genome Editing Tools for the Treatment of Hyperlipidemia

Giulio Preta  1   2
Affiliations
Review

Development of New Genome Editing Tools for the Treatment of Hyperlipidemia

Giulio Preta. Cells. .

Abstract

Hyperlipidemia is a medical condition characterized by high levels of lipids in the blood. It is often associated with an increased risk of cardiovascular diseases such as heart attacks and strokes. Traditional treatment approaches for hyperlipidemia involve lifestyle modifications, dietary changes, and the use of medications like statins. Recent advancements in genome editing technologies, including CRISPR-Cas9, have opened up new possibilities for the treatment of this condition. This review provides a general overview of the main target genes involved in lipid metabolism and highlights the progress made during recent years towards the development of new treatments for dyslipidemia.

Keywords: CRISPR-Cas9; LDL cholesterol; PCSK9; base editing; cardiovascular disease.

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Conflict of interest statement

The author declares no conflict of interests.

Figures

Figure 1
Figure 1
Main therapies used for the treatment of hyperlipidemia with the associated mechanism of action. Different drugs are indicated in red, and the arrows point to the specific molecular targets. A few compounds can act at different levels. Figure modified from [9], licensed under CC-BY 4.0.
Figure 2
Figure 2
Differences between the CRISPR-Cas9 and base editors methodologies. Traditional CRISPR-Cas9 gene editing (left panel) introduces double-strand breaks, which can lead to off-target effects. Base editing (right panel) avoids double-strand breaks due to catalytically inactive Cas9 (dCas9), thereby limiting the occurrence of off-target effects.
See this image and copyright information in PMC

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