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Updated: Jul 16, 2025
09:51
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
Published on: May 25, 2018
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Drug delivery systems for CRISPR-based genome editors
Victoria Madigan1,2,3,4,5, Feng Zhang1,2,3,4,5, James E Dahlman6
1Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Nature Reviews. Drug Discovery
|September 18, 2023
Summary
CRISPR gene editing drugs require effective delivery systems to enter cells safely. This review covers adeno-associated viruses and lipid nanoparticles, examining their engineering, preclinical, and clinical applications for CRISPR delivery.
Area of Science:
- Biotechnology
- Genetics
- Drug Delivery
Background:
- CRISPR-based therapeutics offer precise genetic manipulation.
- Efficient and safe delivery of CRISPR systems into target cells remains a significant challenge.
- Immune responses and off-target delivery necessitate sophisticated delivery vehicles.
Purpose of the Study:
- To review current drug delivery systems for CRISPR-based genome editors.
- To focus on adeno-associated viruses and lipid nanoparticles.
- To discuss future preclinical targeting strategies for CRISPR delivery.
Main Methods:
- Review of engineered adeno-associated viruses and lipid nanoparticles.
- Analysis of preclinical animal model characterization data.
- Highlighting data from recent clinical trials of CRISPR delivery systems.
- Discussion of preclinical targeting using polymers, proteins, and virus-like particles.
Main Results:
- Adeno-associated viruses and lipid nanoparticles are primary delivery systems for CRISPR technology.
- Engineering and characterization in preclinical models inform clinical translation.
- Early clinical trial data provide insights into the safety and efficacy of these systems.
- Diverse preclinical strategies show promise for future CRISPR delivery.
Conclusions:
- Delivery systems are critical for the clinical success of CRISPR-based therapies.
- Adeno-associated viruses and lipid nanoparticles are established but evolving delivery platforms.
- Ongoing research into novel delivery vehicles will expand therapeutic options for genetic diseases.
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