Perspective: Hepatocyte-Directed Base Editing as Novel Treatment for Human Dyslipidemia—Current Status and Remaining Challenges
Hyperlipidemia is a major risk factor for the development of atherosclerotic cardiovascular disease. Lipid-lowering drug therapies therefore still form the heart of the ongoing battle against the occurrence of cardiovascular events. However, in light of the important improvements in gene interferenc...
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| Published in | Arteriosclerosis, thrombosis, and vascular biology Vol. 43; no. 6; pp. 832 - 835 |
|---|---|
| Main Authors | , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Lippincott Williams & Wilkins
01.06.2023
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| Subjects | |
| Online Access | Get full text |
| ISSN | 1079-5642 1524-4636 1524-4636 |
| DOI | 10.1161/ATVBAHA.122.318354 |
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| Abstract | Hyperlipidemia is a major risk factor for the development of atherosclerotic cardiovascular disease. Lipid-lowering drug therapies therefore still form the heart of the ongoing battle against the occurrence of cardiovascular events. However, in light of the important improvements in gene interference and editing that have been made during the last 2 decades, gene therapy—the genetic modification of cells to produce a permanent therapeutic effect—is currently employed to relief hypercholesterolemic subjects from their potential (chronic) cardiovascular disease burden. In this perspective, we review the current status regarding hepatocyte-directed base editing to treat human dyslipidemia and provide suggestions for further technological improvement. |
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| AbstractList | Hyperlipidemia is a major risk factor for the development of atherosclerotic cardiovascular disease. Lipid-lowering drug therapies therefore still form the heart of the ongoing battle against the occurrence of cardiovascular events. However, in light of the important improvements in gene interference and editing that have been made during the last 2 decades, gene therapy-the genetic modification of cells to produce a permanent therapeutic effect-is currently employed to relief hypercholesterolemic subjects from their potential (chronic) cardiovascular disease burden. In this perspective, we review the current status regarding hepatocyte-directed base editing to treat human dyslipidemia and provide suggestions for further technological improvement. Hyperlipidemia is a major risk factor for the development of atherosclerotic cardiovascular disease. Lipid-lowering drug therapies therefore still form the heart of the ongoing battle against the occurrence of cardiovascular events. However, in light of the important improvements in gene interference and editing that have been made during the last 2 decades, gene therapy-the genetic modification of cells to produce a permanent therapeutic effect-is currently employed to relief hypercholesterolemic subjects from their potential (chronic) cardiovascular disease burden. In this perspective, we review the current status regarding hepatocyte-directed base editing to treat human dyslipidemia and provide suggestions for further technological improvement.Hyperlipidemia is a major risk factor for the development of atherosclerotic cardiovascular disease. Lipid-lowering drug therapies therefore still form the heart of the ongoing battle against the occurrence of cardiovascular events. However, in light of the important improvements in gene interference and editing that have been made during the last 2 decades, gene therapy-the genetic modification of cells to produce a permanent therapeutic effect-is currently employed to relief hypercholesterolemic subjects from their potential (chronic) cardiovascular disease burden. In this perspective, we review the current status regarding hepatocyte-directed base editing to treat human dyslipidemia and provide suggestions for further technological improvement. |
| Author | Hoekstra, Menno Van Eck, Miranda Van Berkel, Theo J.C. |
| AuthorAffiliation | Division of BioTherapeutics (M.H., M.V.E., T.J.C.V.B.), Leiden Academic Centre for Drug Research, Leiden University, The Netherlands |
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| SubjectTerms | Cardiovascular Diseases - drug therapy Cardiovascular Diseases - therapy Dyslipidemias - drug therapy Dyslipidemias - genetics Gene Editing Hepatocytes Humans Hypolipidemic Agents - therapeutic use |
| Title | Perspective: Hepatocyte-Directed Base Editing as Novel Treatment for Human Dyslipidemia—Current Status and Remaining Challenges |
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