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Delivery of Proteins, Peptides or Cell-impermeable Small Molecules into Live Cells by Incubation with the Endosomolytic Reagent dfTAT
Published on: September 2, 2015
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A peptide delivery system sneaks CRISPR into cells.
Xingang Guan1,2, Zhimin Luo1,3, Wujin Sun4
1From the Department of Bioengineering, Center for Minimally Invasive Therapeutics at California Nanosystems Institute, UCLA, Los Angeles, California 90095.
The Journal of Biological Chemistry
|November 4, 2018
Summary
Researchers developed a new peptide delivery method for CRISPR-Cas9 genome editing. This approach successfully disrupted genes in cells and mice, offering potential therapeutic strategies for metabolic diseases like obesity and diabetes.
Area of Science:
- Molecular Biology
- Gene Editing Technologies
- Biotechnology
Background:
- CRISPR-Cas9 is a precise genome editing tool but faces delivery challenges.
- Efficient delivery is crucial for CRISPR-Cas9's clinical applications.
Purpose of the Study:
- To develop and evaluate a novel peptide-based delivery system for CRISPR-Cas9 ribonucleoprotein complexes.
- To demonstrate the efficacy of this delivery system in disrupting target genes in vitro and in vivo.
Main Methods:
- Utilized an amphipathic peptide for delivering Cas9/sgRNA ribonucleoprotein complexes.
- Assessed gene disruption efficiency by targeting GFP genes in cells and mice.
- Investigated the functional consequences of gene disruption in pre-adipocytes.
Main Results:
- The peptide successfully delivered Cas9/sgRNA complexes, leading to targeted gene disruption.
- Demonstrated successful gene editing in both cellular and animal models.
- Disruption of the Nrip1 gene induced a "browning" phenotype in pre-adipocytes.
Conclusions:
- The developed amphipathic peptide is an effective delivery vehicle for CRISPR-Cas9 genome editing.
- This delivery method shows promise for therapeutic applications, particularly in metabolic disorders.
- The study opens new avenues for tackling obesity and diabetes through precise gene editing.
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