Development of Gene Therapy Using eToeholds
Gene therapy refers to the implantation of normal genes into target cells to replace genetically defective genes in patient cells or to turn off and inhibit abnormally expressed genes to achieve the purpose of preventing and treating diseases. It is a clinical medical technology that is widely used in genetic diseases, malignant tumors, cancer, and other diseases. The target cells for gene therapy can be divided into somatic cells and germ cells. Introducing genes into somatic cells can work effectively for a long time. The modification of germ cells involves numerous technical operations and ethical issues, so it is not a treatment method.
Conventional Gene Therapies
Gene editing can replace or correct the wrong gene sequence to restore the DNA in the cell to a normal state; or introduce normal genes, enhance the expression of normal genes through non-site-directed integration, and make up for the function of problematic genes. However, the disadvantage is that off-target effects may occur. Appropriate gene vectors can also be selected to package exogenous genes, and then fused with target cells or directly injected into diseased tissues for synthetic expression. However, the disadvantage is that it contains few exogenous genes, has poor stability, can cause immune system responses, and even activate the proto-oncogenes of cells.
Eukaryotic Toehold Switches in Gene Therapy Development
Because of the dangers of gene therapy when transgenes are expressed in the wrong cells, it is important to find a way to reduce the off-target effects of gene therapy. Eukaryotic Toehold Switches (eToeholds) are biological elements built into RNA that express linked protein-coding sequences only when cell-specific or viral RNA is present. This feature enables eToeholds to differentiate between different types of cells (including human cells), and only produce the required proteins and function accordingly in those cells that cause or are affected by a particular disease. It has been shown that cancer cells can be identified by detecting eToeholds of tyrosinase mRNA, and when cancerous cells are detected, toxic proteins are produced in these cells, triggering cell death. At CD BioSciences, we have a professional eToeholds technology platform and research team, which can provide eToeholds-based solutions and technical support for scholars engaged in gene therapy research.
The Services We Can Provide Based on eToeholds
eToeholds detect specific mRNA sequences in cells and trigger the production of specific proteins. The eToeholds device offers multiple opportunities for more targeted RNA therapeutics, in vitro cell and tissue engineering approaches and sensing a variety of biological threats to humans and other higher organisms. Using the established eToeholds technology platform, CD BioSciences can synthesize an eToeholds RNA molecule carrying therapeutic, diagnostic, or other functions for researchers engaged in gene therapy development. The molecule is delivered to the cell and can produce the desired protein in the diseased cell and perform the corresponding function. The basic services we can provide are as follows.
Advantages of eToeholds in Gene Therapy Development
- eToeholds can accurately and efficiently identify target cells.
- eToeholds can dramatically reduce off-target effects in gene therapy.
- eToeholds have a higher safety profile and can reduce the side effects of targeted RNA therapy.
CD BioSciences looks forward to providing you with eToeholds-based technical support in gene therapy-related research. If you are interested in the application of eToeholds in gene therapy, please feel free to contact us.
References
- Green, A.A.; et al. Toehold switches: de-novo-designed regulators of gene expression. Cell. 2014, 159(4): 925-939.
- Zhao, E.M.; et al. RNA-responsive elements for eukaryotic translational control [published online ahead of print, 2021 Oct 28]. Nat Biotechnol. 2021, 10.1038/s41587-021-01068-2.