Nat Commun May 2023 PMID 37188660

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy.

Lisa N Kasiewicz, Souvik Biswas, Aaron Beach, Huilan Ren, Chaitali Dutta, Anne Marie Mazzola, Ellen Rohde, Alexandra Chadwick, Christopher Cheng, Sara P Garcia, Sowmya Iyer, Yuri Matsumoto, Amit V Khera, Kiran Musunuru, Sekar Kathiresan

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Mentions

Peak: 6 in May 2023

across 4 sources since May 2023

Extended reach

4,180

potential audience

Total likes

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Mentions per month

May 2023 – Nov 2023

Where it's discussed

X / Twitter7 posts
News2 articles
Blogs2 articles
YouTube1 videos

AI synopsis

What people are saying

Many discussions emphasize the breakthrough nature of the delivery system, with phrases like 'key' and 'promising' suggesting optimism. The repeated references to improvements in gene editing efficiency and independence from LDL receptors show that users are deeply engaged with the technical implications and potential real-world impact of this research.

AgreementModerate agreement

Most discussions acknowledge the significance of the delivery method and the potential of GalNAc-Lipid nanoparticles, showing general support.

InterestHigh interest

The topics attract high interest due to their relevance to gene therapy and innovative delivery techniques, as reflected in enthusiastic language.

EngagementModerate engagement

Comments cite specific details, such as improved liver gene editing and independence from LDL receptors, indicating a moderate level of engagement.

ImpactHigh impact

The discussions highlight the transformative potential of this technology for treatments, emphasizing its importance for various patient groups and future therapies.

Abstract in brief

GalNAcLipid nanoparticles have been designed to deliver CRISPR base editing therapies to the liver independently of the LDL receptor, which is crucial for patients with conditions like homozygous familial hypercholesterolemia.
In studies involving mice and nonhuman primates, adding an optimized GalNAc-based ligand to the nanoparticles significantly increased liver gene editing (from 5% to 61%) and reduced off-target effects, showing promise for more effective treatment.
The therapy demonstrated durable results, with up to 89% reduction in blood ANGPTL3 protein lasting six months, indicating that this approach could benefit both patients with normal LDL receptor function and those with receptor deficiencies.]

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All mentions

Non-LDLR Dependent Hepatic Delivery of CRISPR Therapy Using GalNAc Lipid Nanoparticles

GalNAc-Lipid nanoparticles enable delivery of CRISPR base editing therapies to the liver independently of the LDL receptor, crucial for treating conditions like homozygous familial hypercholesterolemia.

STEM RTCL TV · Aug 17, 2023 96 views

biobio

@GudivadaBalu · X

@RNAiAnalyst I think delivery is key.. https://t.co/4lQbo2QeQT

Nov 13, 2023 · 1 like · 124 followers View post →

ColSci

@colsci_papers · X

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy https://t.co/mEY45dqPJR

May 25, 2023 · 402 followers View post →

KanzakiS

@nchdaks · X

RT @ScienceCrisp: "GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a #CRISPR base editing therapy" Kasiewicz LN […

May 17, 2023 · 1 share · 677 followers View post →

CRISP_SCIENCE

@ScienceCrisp · X

May 17, 2023 · 1 like · 1 share · 1,917 followers View post →

CRISPR Articles

@CRISPR_JC · X

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy. https://t.co/XAxUwKaWwN

May 16, 2023 · 1 like · 38 followers View post →

LUKMAN AFOLABI

@saintlukman · X

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy https://t.co/ZfU4TrMhIn https://t.co/VJi4p8Al1F

May 16, 2023 · 922 followers View post →

Purebiotech

@Purebiotech14 · X

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy https://t.co/zFs5OajI3P

May 15, 2023 View post →