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Updated: Jul 20, 2025
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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
Published on: August 2, 2018
8.2K
Gene editing therapeutics based on mRNA delivery
Juliana Popovitz1, Rohit Sharma2, Reyhane Hoshyar1
1GenEdit, 681 Gateway Blvd., South San Francisco, CA 94080, USA.
Advanced Drug Delivery Reviews
|July 29, 2023
Summary
Gene editing therapeutics using mRNA offer new ways to treat genetic disorders. This review covers mRNA delivery, gene editing technologies, and future advancements in CRISPR and base editing systems.
Area of Science:
- Molecular Biology
- Biotechnology
- Genetic Engineering
Background:
- Gene editing technologies like CRISPR-Cas, base editors, and other systems show significant therapeutic promise.
- These technologies offer potential treatments for previously untreatable genetic disorders.
Purpose of the Study:
- To review the therapeutic applications of gene editing technologies utilizing messenger RNA (mRNA) delivery.
- To discuss advancements in gene editing technology engineering and improvement.
Main Methods:
- Focus on mRNA delivery for gene editing applications.
- Examination of both ex vivo and in vivo gene editing strategies.
- Review of next-generation CRISPR and base editing systems.
Main Results:
- mRNA delivery is a viable method for gene editing therapeutics.
- Engineering and improvements are enhancing gene editing technology efficacy.
- Ex vivo and in vivo approaches demonstrate diverse therapeutic potentials.
Conclusions:
- mRNA-based gene editing holds substantial promise for treating genetic diseases.
- Ongoing advancements in gene editing technologies, including CRISPR and base editors, are expanding therapeutic possibilities.
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