Publication Date
12-8-2022
Journal
Molecular Therapy Methods & Clinical Development
DOI
10.1016/j.omtm.2022.10.009
PMID
36381302
PMCID
PMC9630778
PubMedCentral® Posted Date
10-13-2022
PubMedCentral® Full Text Version
Post-print
Published Open-Access
no
Abstract
Lipoprotein(a) (Lp(a)) represents a unique subclass of circulating lipoprotein particles and consists of an apolipoprotein(a) (apo(a)) molecule covalently bound to apolipoprotein B-100. The metabolism of Lp(a) particles is distinct from that of low-density lipoprotein (LDL) cholesterol, and currently approved lipid-lowering drugs do not provide substantial reductions in Lp(a), a causal risk factor for cardiovascular disease. Somatic genome editing has the potential to be a one-time therapy for individuals with extremely high Lp(a). We generated an
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