{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Fitzgerald K"],"funding":["NHLBI NIH HHS","NIH"],"pagination":["60-68"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC4387547"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["383(9911)"],"pubmed_abstract":["Background
Proprotein convertase subtilisin/kexin type 9 (PCSK9) binds to LDL receptors, leading to their degradation. Genetics studies have shown that loss-of-function mutations in PCSK9 result in reduced plasma LDL cholesterol and decreased risk of coronary heart disease. We aimed to investigate the safety and efficacy of ALN-PCS, a small interfering RNA that inhibits PCSK9 synthesis, in healthy volunteers with raised cholesterol who were not on lipid-lowering treatment.Methods
We did a randomised, single-blind, placebo-controlled, phase 1 dose-escalation study in healthy adult volunteers with serum LDL cholesterol of 3·00 mmol/L or higher. Participants were randomly assigned in a 3:1 ratio by computer algorithm to receive one dose of intravenous ALN-PCS (with doses rangi"],"journal":["Lancet (London, England)"],"pubmed_title":["Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial."],"pmcid":["PMC4387547"],"funding_grant_id":["HL020948","P01 HL020948"],"pubmed_authors":["Mant T","Frank-Kamenetsky M","Nochur SV","Cehelsky J","Sutherland JE","Karsten V","Clausen VA","Gollob JA","Chiesa J","Simon A","Vaishnaw AK","Horton J","Liebow A","Bettencourt BR","Hutabarat RM","Fitzgerald K","Ritter J","Kotelianski V","Munisamy M","Shulga-Morskaya S"],"additional_accession":[]},"is_claimable":false,"name":"Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial.","description":"Background
Proprotein convertase subtilisin/kexin type 9 (PCSK9) binds to LDL receptors, leading to their degradation. Genetics studies have shown that loss-of-function mutations in PCSK9 result in reduced plasma LDL cholesterol and decreased risk of coronary heart disease. We aimed to investigate the safety and efficacy of ALN-PCS, a small interfering RNA that inhibits PCSK9 synthesis, in healthy volunteers with raised cholesterol who were not on lipid-lowering treatment.Methods
We did a randomised, single-blind, placebo-controlled, phase 1 dose-escalation study in healthy adult volunteers with serum LDL cholesterol of 3·00 mmol/L or higher. Participants were randomly assigned in a 3:1 ratio by computer algorithm to receive one dose of intravenous ALN-PCS (with doses rangi","dates":{"release":"2014-01-01T00:00:00Z","publication":"2014 Jan","modification":"2026-05-01T15:16:18.361Z","creation":"2019-03-27T01:49:31Z"},"accession":"S-EPMC4387547","cross_references":{"pubmed":["24094767"],"doi":["10.1016/S0140-6736(13)61914-5","10.1016/s0140-6736(13)61914-5"]}}