Genome editing based on programmable nucleases (CRISPR-Cas systems) overcoming the imprecision of current gene therapy is likely to become the next-generation gene therapy technology. At present, there are four major classes of engineered nucleases: mega nucleases, zinc finger nucleases (ZFNs), transcription activator–like effector nucleases (TALENs) and the CRISPR-Cas system.
CRISPR-Cas9 has been widely used in the research field as well as in disease treatment. In recent years, CRISPR-Cas9 has quickly progressed to the clinical stage for the treatment.
More and more advanced systems are coming out every year. But before using such technologies, they must be thoroughly tested. Certain types of systems show good performance for certain parts of the genome or tissues. We believe that such testing should be carried out for each case separately due to the complexity of the genome structure and the irreparable consequences in case of an error.
To solve this problem, it is necessary to create a universal cellular biosensor that can determine the activity of cas systems in cells quickly and without additional complex manipulations such as sequencing. At present, there are four major classes of engineered nucleases: mega nucleases, zinc finger nucleases (ZFNs), transcription activator–like effector nucleases (TALENs) and the CRISPR-Cas system.
The first task we undertake is the analysis of the performance of the most popular genome editing systems based on literature data in order to understand the strengths and limitations of these systems. This information serves as a foundation for our subsequent work.
Next, we move on to the selection of the main components of the genetic editing system. These carefully chosen components are instrumental in developing a biosensor that enables us to detect and monitor the activity of the genetic editing system.
Once the components are selected, the next task at hand is the design of a molecular biosensor system. This system operates based on genetic chains, allowing us to specifically monitor the activity of individual components within the genetic editing system.
Moving forward, we focus on creating a genetic chain capable of detecting the activity of the most popular editing systems.
Lastly, we plan to evaluate the activity of the obtained system in vivo, employing model cellular systems. By conducting experiments within these controlled environments, we can observe and measure the performance of our genetic editing system, further refining and optimizing its functionality.
The use of genetic editing technologies will solve many problems related to the treatment of genetic diseases. However, the use of such technologies is causing serious debate in society. Systems like ours will allow us to convey to society the idea of the safety and stability of such systems and will also be called to solve problems related to the selection of the most optimal editing system for specific cases. We also want to provide a comprehensive solution consisting not only of a biosensor, but also a model capable of predicting the behavior of a genetic editing system.
Moreover, the popularization of the ideas of synthetic biology is always one of our primary goals. We want to explain to society all the positive aspects of such technologies as CRISPR-Cas and to urge about the real threats that are hidden in these technologies.
We have developed a simple system for assembling and confirming the activity of various CRISPR-Cas systems. Our system will help you quickly scan potential targets and select the best options for CRISPR-Cas systems to interact with them. In our opinion, such a system will speed up the process of choosing the most effective system for future therapy, as well as reduce the possible harm of the usage of such technologies.
Finally, here is our promotional video!