Kiran Musunuru

This study corrects a common pathogenic variant in MMA using adenine base editing in hepatocytes, showing efficient in vitro editing with minimal off-target effects.

This study develops anti-OxLDL CAR Tregs that reduce atherosclerotic plaque formation in mice by targeting OxLDL-mediated inflammation.

This study evaluates hybrid guide RNAs to improve the specificity and efficiency of adenine base editing therapies for genetic disorders.

The paper generates gene expression data from iPSC-derived cardiomyocytes in a multiethnic cohort and makes the results publicly available.

This study evaluates the durability of epigenome editing for reducing gene expression in cardiovascular disease targets, finding durable effects for PCSK9 but not for ANGPTL3 or AGT.

The paper identifies cell-type specific splicing quantitative trait loci (sQTLs) in iPSCs and HLCs and shows they contribute to lipid level variation beyond what is captured by gene expression alone.

This study develops GalNAc-LNPs for liver gene editing in NHPs, achieving high editing efficiency and ANGPTL3 reduction in both HoFH and wild-type models.

ONE-seq identifies population-specific off-target effects of CRISPR gene editors using global human genome sequence diversity.

The paper identifies a missense variant in EDEM3 associated with lower blood triglyceride levels and shows EDEM3 deficiency increases LRP1 expression, reducing plasma TG through enhanced lipoprotein uptake.

This study identifies A1CF as a regulator of plasma triglycerides through posttranscriptional control of VLDL secretion and intracellular stress pathways.