Perspective: Hepatocyte-Directed Base Editing as Novel Treatment for Human Dyslipidemia-Current Status and Remaining Challenges.
Menno HoekstraMiranda Van EckTheo J C Van BerkelPublished in: Arteriosclerosis, thrombosis, and vascular biology (2023)
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.
Keyphrases
- cardiovascular disease
- cardiovascular events
- crispr cas
- current status
- gene therapy
- endothelial cells
- coronary artery disease
- induced pluripotent stem cells
- genome wide
- induced apoptosis
- heart failure
- risk assessment
- pluripotent stem cells
- cardiovascular risk factors
- gene expression
- high fat diet
- cell cycle arrest
- adipose tissue
- climate change
- fatty acid
- risk factors
- cell death
- transcription factor
- genome wide identification