Novel nanoparticles deliver CRISPR gene editing tools into the cell with much higher efficiency

Novel nanoparticles deliver CRISPR gene editing tools into the cell with much higher efficiency

Publication date: Jul 13, 2019

Biodegradable lipid nanoparticles deliver mRNA coding for the gene editing molecules into the cell Credit: From animation by Visual Science and Skoltech, visual-science.

The delivery uses biodegradable synthetic lipid nanoparticles that carry the molecular editing tools into the cell to precisely alter the cells’ genetic code with as much as 90 percent efficiency.

The nanoparticles represent one of the most efficient CRISPR/Cas9 delivery tools reported so far, according to the researchers, and could help overcome technical hurdles to enable gene editing in a broad range of clinical therapeutic applications.

Upon entering the cell, in vitro or in vivo, the linkers are broken and the particles disintegrate for delivery of contents and translation of mRNA into active enzyme for CRISPR/Cas9 genome editing Credit: Qiaobing Xu, Tufts University “We are just starting to see human clinical trials for CRISPR therapies,” said Qiaobing Xu, co-corresponding author of the study and associate professor of biomedical engineering at Tufts University.

information: Ji Liu et al, Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles, Advanced Materials (2019).

DOI: Citation: Novel nanoparticles deliver CRISPR gene editing tools into the cell with much higher efficiency (2019, July 12) retrieved 13 July 2019 from This document is subject to copyright.

Concepts Keywords
Advanced Materials Machinery
Beijing Genetic code
Biodegradable Animation
Biomedical Engineering CRISPR gene editing
Cardiovascular Disease CRISPR-Display
Cas9 Genome editing
Chinese CRISPR
Clinical Trials Cas9
Crispr Immune system
CRISPR Enzymes
Disulfide Bond Biotechnology
Disulfide Bonds Genetic engineering
Duchenne Muscular Dystrophy Life sciences
Enzyme Branches of biology
Fair Dealing Disease cancers
Genetic Code Diseases
Genome Clinical therapeutic applications
Huntington
LDL Cholesterol
Linker
Lipid
Lipids
Liver
Mice
Ming
MRNA
Nanoparticles
Nuclease
Nucleus
Polymers
Protein
Sickle
Tufts University
Viruses
Vivo

Semantics

Type Source Name
gene UNIPROT LARGE1
disease MESH Visual
drug DRUGBANK Coenzyme M
gene UNIPROT SMIM10L2B
gene UNIPROT SMIM10L2A
disease MESH development
gene UNIPROT KIT
pathway BSID Translation
disease MESH sickle cell disease
disease MESH Duchenne muscular dystrophy
disease DOID Duchenne muscular dystrophy
disease MESH cancers
gene UNIPROT PTPN5
gene UNIPROT PCSK9
drug DRUGBANK Cholesterol
disease MESH cardiovascular disease
gene UNIPROT FASTK

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