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mRNA-Based Approaches for Gene Editing

Gene Editing

Apart from the most obvious application for protein replacement therapy, mRNA technology can also be applied in gene editing for expressing the corresponding nucleases. The discovery of the CRISPR-Cas9 (clustered regularly spaced short palindromic repeat-CRISPR-associated protein 9) system, a breakthrough technology, has led to the meteoric rise of the gene-editing field. To date, gene editing has emerged as a new therapeutic option for a wide range of clinical applications, especially for gene-based diseases. Creative Biogene is a leading provider in the mRNA-based drug R&D service. In virtue of mRNA therapeutics' advantages, gene editing is a therapeutic tool to achieve a more vigorous development.

Gene editing

As a therapeutic tool, gene editing enables the alteration of gene expression by introducing site-specific modifications (e.g., correction of deleterious or protective mutations) into the genome of cells. This technology is based on the repairment of a DNA double-stranded break (DSB) in a specific target location of the genome, which is generated by the programmable nuclease, such as CRISPR-Cas, zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs). Since the strategy of target sequence recognition of ZFNs and TALENs is based on DNA -protein interaction, they are limited when creating specific DNA-targeting protein domains for complicated protein engineering. Compared to the above two gene editing nucleases, CRISPR-Cas, one of the RNA-guided DNA endonucleases, accomplishes nuclease targeting through RNA and DNA base pairing, providing a relatively simple manner to alter genomic information. At present, the CRISPR-Cas9 system has become a well-characterized and widely used gene-editing technology. 

mRNA-based approaches for gene editing

Gene Editing

IVT mRNA formulations hold significant potential as vehicles in gene editing, although there are full of challenges. Compared to the other delivery forms of gene editing nucleases, such as protein and pDNA, IVT mRNA has a series of superior advantages. At present, IVT mRNA-based gene-editing nucleases have been mainly used for preclinical studies, involving editing hematopoietic stem and progenitor cells (HSPCs) and T cells in ex vivo. Besides, it is utilized to generate animal models through genome editing of embryos. In addition, for clinical application, mRNA-based gene editing has been achieved in organs, including the liver and lungs, by mainly co-delivering with lipid nanoparticle (LNP) systems.

Advantages of mRNA-based gene editing

  • High efficacy
  • Low off-target effect due to the expression of mRNA is transient
  • Low risk of genome insertion as compared to pDNA form
  • More consistent effect as compared to protein form

Given the rapid expansion of gene editing and the superior advantages of mRNA therapy, we believe the use of mRNA-based gene editing will be significantly increased in the near future. Creative Biogene is one of the leading providers in the services of mRNA-based drug R&D. We are committed to providing our customers with the finest services. Based on the available state-of-the-art facilities and highly experienced staff, we are now offering a series of customized services, but not limited to them, including,

  • mRNA design service
  • Customized mRNA synthesis service
  • A series of mRNA optimization services
  • mRNA delivery vector service

If you would like more information about our services, please don't hesitate to contact us.

References

  1. Sahin, U., et al. (2014). "mRNA-based therapeutics—developing a new class of drugs." Nature reviews Drug discovery, 13(10), 759-780.
  2. Gómez-Aguado, I., et al. (2020). "Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives." Nanomaterials, 10(2), 364.

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