Tiny Lipids as Delivery Vehicle May Make CRISPR-Cas9 More Effective Gene Editing Tool

Tiny Lipids as Delivery Vehicle May Make CRISPR-Cas9 More Effective Gene Editing Tool

Publication date: Aug 27, 2019

This post was originally published on this site Tiny fatty (lipid) particles can be used to enhance the delivery of gene editing tools, such as CRISPR-Cas9, to targeted cells and considerably improve their ability to possibly treat human disorders like Huntington’s disease, researchers reported. By delivering the Cas9 enzyme and appropriate ribonucleic acids (RNAs) into a cell, an organism’s genome can be cut at any desired location, like a tailor-made gene modification. Researchers at Tufts University and the Chinese Academy of Sciences engineered lipid nanoparticles that can be loaded with the Cas9 enzyme messenger RNA (mRNA) encoding sequence (much like an instructions manual) plus an engineered ‘single-guide’ RNA molecule (sgRNA). In the laboratory, this new system of simultaneous delivery of the Cas9 sequence and sgRNA molecule achieved up to 90% efficacy within 24 hours, -which is a significant enhancement” compared with other delivery systems, the researchers noted. They also tested the efficacy of their new system in mice, seeking to lower the amount of the PCSK9 gene, which encodes a protein that regulates cholesterol levels in the bloodstream and can reduce the risk of cardiovascular disease.

Concepts Keywords
Adenine Considerable potential applications
Advanced Materials Disorders likeHuntingtons disease
Biocompatibility Cut
Blood System treat disorders
Cardiovascular Disease Diseases
Cas9 Branches of biology
Chinese Life sciences
Cholesterol Genetic engineering
CRISPR Genome editing
Cytosine Biotechnology
DNA Immune system
Duchenne Muscular Dystrophy Enzymes
Enzyme Cas9
Gene CRISPR
Genetic CRISPR gene editing
Genome Human Nature
Germline
Guanine
Intravenously
Lipid
Liver
Mice
Ming
Molecule
MRNA
Nanoparticles
Sickle
Tailor
Viral Vectors

Semantics

Type Source Name
gene UNIPROT SLC35G1
gene UNIPROT DESI1
drug DRUGBANK Adenine
drug DRUGBANK Guanine
gene UNIPROT PCSK9
drug DRUGBANK Cholesterol
disease MESH cardiovascular disease
pathway BSID Release
disease MESH sickle cell disease
disease MESH Duchenne muscular dystrophy
disease DOID Duchenne muscular dystrophy
disease MESH cancers
gene UNIPROT PTPN5

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