Translation. Region: Russian Federation –
Source: Peoples'Friendship University of Russia
An important disclaimer is at the bottom of this article.
The 12th Russian Biotechnology Forum, OpenBio, took place at the end of September. This major event brings together representatives of science, business, and government to address the challenges of ensuring the sustainable development of the biotechnology industry and the national economy. The program included expert discussions, roundtables, presentations by industry leaders, master classes, and technology and equipment presentations.
Following the three days of work, the scientific jury recognized the best papers in each section. One of the winners in the "Biotechnology" section was Alena Borisova, a second-year master's student at the RUDN University Institute of Medicine (Gene and Tissue Engineering program) and a researcher at the Institute of Gene Biology of the Russian Academy of Sciences. At the forum, she presented a paper titled "Creation of an Isogenic Cell Model Using CRISPR/Cas9 to Assess CFTR Ion Channel Function."
We spoke with Alena to find out what the most popular topics were at the forum, the relevance of her work, and her plans for future research. We also asked her for advice for schoolchildren and students on how to succeed in biotechnology.
What key trend or challenge in the biotech industry was the leitmotif of this year's OpenBio forum? What was the most frequently discussed topic?
The forum's leitmotif was the strategic development of two interconnected areas: the transition from scientific discoveries to their accessible and large-scale application, and strengthening the country's scientific and technological sovereignty. The emphasis shifted from the innovations themselves to their practical implementation. The key challenge today is not simply creating a breakthrough technology, but establishing its effective implementation in production and ensuring its widespread availability to the economy and society.
This trend was evident in scientists' presentations on developments in the field of future medicine. Increasingly, the focus is not on finding a cure for a single disease, but on creating universal platforms that can be used to quickly develop drugs for a variety of purposes. Prominent examples of such platforms, which were actively discussed, include mRNA technologies (which everyone became aware of thanks to the COVID-19 vaccines).
At the same time, other innovative areas are rapidly developing: gene and cell therapy and new drug delivery systems. These open up new possibilities in the fight against oncological, autoimmune, infectious, and hereditary diseases. Adapting the regulatory framework has also become an important part of the dialogue, as existing regulations must keep pace with the rapid development of such innovations, ensuring they reach patients more quickly.
Tell us about your report and project. What is an "isogenic cell model" and what is it applicable for? What were you able to discover using the model you created, and what are the next steps in this research?
As part of a project at the Institute of Gene Biology of the Russian Academy of Sciences, my colleagues and I are working to create 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 universal treatment for this disease, the search for new therapeutic approaches is extremely urgent, and this requires adequate laboratory models that allow for the initial screening of potential drugs.
Our goal is to create a universal tool capable of accelerating the development of treatments for patients who currently remain untreated. To do this, we used the CRISPR/Cas9 genome editing method to create what's called an isogenic cell model. Essentially, this is a pair of cell lines that are genetically identical in every way except for one altered region—in our case, the CFTR gene. We took healthy cells and "turned off" this gene, creating a system: 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 response will be associated specifically with the target mutation, rather than the overall genetic background. This significantly increases the accuracy of experiments. Furthermore, our model can be used for fundamental studies of disease pathogenesis and CFTR protein function.
In addition to creating the cell line itself, we developed a functional assay based on it to evaluate CFTR protein function in cells. CFTR normally functions as an ion channel, responsible for transporting chloride ions and maintaining water balance in tissues. To visualize the consequences of its absence, we grew three-dimensional structures from the cells—miniature replicas of organs. Healthy cells formed structures with an internal cavity, while cells with cystic fibrosis formed only dense spheroids without a cavity. When we activated CFTR by adding a special substance to our model, the healthy structures began to swell and increase in volume, as their cells were able to transport chloride ions and water into the internal cavity. This response was not observed in cells with a defective CFTR, as this transport mechanism was impaired. Thus, our system allows for direct observation of the physiological consequences of cystic fibrosis-related disorders in the laboratory.
We plan to further validate the resulting model using therapeutic agents to confirm its clinical significance. After that, we plan to actively use it to screen new potential drugs for cystic fibrosis.
What advice would you give to schoolchildren or students who would like to connect their lives with modern biotechnology?
First of all, I want to emphasize: modern biotechnology requires broad interdisciplinary knowledge. The most interesting discoveries today are born at the intersection of sciences. Knowing only biology or chemistry is no longer enough. A good biotechnologist is a specialist with deep knowledge in one field and a broad perspective in related ones. Therefore, I advise schoolchildren and students to love biology, but not to forget about mathematics, physics, and chemistry, and not to neglect "non-core" subjects. And it's also essential to be familiar with IT—the ability to work with data has become the new superpower of modern scientists. And yes, English is a gateway to the global scientific community; you can't get anywhere without it.
It's also helpful to develop soft skills: participate in public speaking, try your hand at project work, and actively network. Case competitions are a great opportunity for students to do this. The ability to work in a team and communicate your ideas is just as important as conducting a successful experiment!
Based on my experience, I also recommend immersing yourself in a real scientific environment as early as possible, starting lab internships as early as your first year. This way, you'll not only be able to apply your knowledge in practice but also truly understand the purpose of all those complex topics covered in class. When you encounter a real scientific problem and see how theory works in a real experiment, the whole picture finally comes together. It's also important to keep up with trends in your field of interest—reading scientific literature and familiarizing yourself with the latest research.
But the most valuable advice, in my opinion—and it's useful not only for future biotechnologists—is to take advantage of every opportunity for development offered by school, university, and even life itself. And to seek them out yourself! You never know what might be useful in the future, but every experience makes us stronger.
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