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After three days of work, the scientific jury selected the best reports from each section. One of the winners in the Biotechnology section was Alyona Borisova, a second-year master’s student at the RUDN Institute of Medicine and an employee at the Institute of Gene Biology of the Russian Academy of Sciences. At the forum, she presented her research on ‘Creating an Isogenic Cell Model Using CRISPR/Cas9 to Assess the Function of the CFTR Ion Channel’.

We spoke with Alyona to find out about the most popular topics at the forum, the relevance of her work, and her plans for future research. We also asked her for advice on how to succeed in the field of biotechnology.

What was the key trend or challenge in the biotechnology industry that became the leitmotif of this year’s OpenBio forum? What was the most discussed topic?

The leitmotif of the forum was the strategic development of two interconnected areas: the transition from scientific discoveries to their accessible and widespread application, and the strengthening of the country’s scientific and technological sovereignty. The focus shifted from the innovation itself to its practical implementation. The key challenge today is not just to create breakthrough technology, but also to ensure its effective implementation in production and make it widely available for the economy and society.

This trend was evident in the presentations by scientists who focused on future medical developments. Instead of focusing on finding a cure for a specific disease, the focus shifted to creating universal platforms that can be quickly adapted for various purposes. mRNA technologies, which have gained significant attention due to their use in COVID-19 vaccines, are a notable example of such platforms.

In parallel, other innovative areas are also actively developing, including gene and cell therapy, as well as new drug delivery systems. These advancements offer new possibilities for treating cancer, autoimmune, infectious, and hereditary diseases. The dialogue also focused on adapting the regulatory framework to keep pace with the rapid development of these innovations, ensuring that they can reach patients more quickly.

Tell us about your report and project. What is an “isogenic cell model” and how is it used? What have you discovered using this model, and what are the next steps in your research?

As part of a project at the Institute of Gene Biology of the Russian Academy of Sciences, my colleagues and I are working on creating a convenient and relevant cell model of cystic fibrosis, a severe hereditary disease caused by mutations in the CFTR gene. Since there is currently no cure for this disease, the search for new therapeutic approaches is crucial, and this requires the use of appropriate laboratory models that can be used for initial screening of potential drugs.

Our goal is to create a universal tool that can accelerate the development of therapies for patients who currently lack treatment. To achieve this, we have used the CRISPR/Cas9 genome editing technique to create an isogenic cell model. Essentially, this is a pair of cell lines that are genetically identical in all aspects, except for a single altered region, in our case, the CFTR gene. We have taken healthy cells and “turned off” this gene, resulting in a system where one line serves as a healthy control and the other as a disease model.

The key advantage of this approach is that any observed differences in drug responses will be attributed to the targeted mutation rather than the overall genetic background. This significantly enhances the accuracy of our experiments. Additionally, our model can be used for fundamental studies on the pathogenesis of the disease and the function of the CFTR protein.

In addition to creating the cell line itself, we have developed a functional test based on it that allows us to evaluate the function of the CFTR protein in cells. Normally, CFTR acts as an ion channel responsible for transporting chloride ions and maintaining water balance in tissues. To visualize the consequences of its absence, we have grown three-dimensional structures from the cells, which are miniature representations of organs. Healthy cells formed structures with a cavity inside, while cells with cystic fibrosis formed only dense spheroids without a cavity. When we activated CFTR by adding a specific substance to our model, the healthy structures began to swell and increase in size, as their cells were able to transport chloride ions and water into the internal cavity. However, in the case of cells with a broken CFTR, this response was not observed, as their transport mechanism was impaired. Thus, our system allows us to directly observe the physiological consequences of the abnormalities caused by cystic fibrosis in the laboratory.

In the future, we plan to validate the model using therapeutic agents to confirm its clinical significance. After that, we plan to actively use it for screening new potential drugs against cystic fibrosis.

What would you advise schoolchildren or students who would like to pursue a career in modern biotechnology?

First of all, I would like to emphasize that modern biotechnology requires a broad interdisciplinary knowledge. Today, the most interesting discoveries are made at the intersection of different sciences. It is no longer enough to know only biology or only chemistry. A good biotechnologist is a specialist with in-depth knowledge in one field and a broad perspective in related areas. Therefore, I would advise schoolchildren and students to love biology, but also not to forget about mathematics, physics, and chemistry. It is also important to be friends with IT, as the ability to work with data has become a new superpower for modern scientists. Additionally, English is a key to joining the global scientific community, and it is essential to have a good command of the language. It is also beneficial to develop soft skills, such as participating in public presentations, trying your hand at project work, and actively engaging in networking. Various case competitions provide excellent opportunities for students to do so. The ability to work in a team and communicate effectively is just as important as conducting a successful experiment!

Based on my experience, I would also recommend immersing yourself in the real scientific environment as early as possible, starting internships in laboratories during your first years of university. This not only allows you to apply your knowledge in practice, but also helps you truly understand the purpose of all the complex topics covered in your classes. When you encounter a real scientific challenge and see how theory works in a real experiment, the whole picture begins to make sense. It is also important to stay up-to-date with the latest trends in your field of interest by reading scientific literature and familiarizing yourself with the latest research.

In my opinion, the most valuable advice is not only for future biotechnologists: you should use every opportunity for development that school, university, and life offer. And you should look for them yourself! You never know what will be useful in the future, but every experience makes us stronger.