Using ancestral protein reconstruction techniques, the team studies molecules that existed millions of years ago in order to identify biological properties that are no longer found in present-day organisms.
Every year on 21 June, World Amyotrophic Lateral Sclerosis (ALS) Day is observed. ALS is a neurodegenerative disease that affects the motor neurons responsible for controlling voluntary movement. In Spain, around 4,000 people are currently living with this condition, which is characterised by a progressive loss of mobility that has a profound impact on the autonomy and quality of life of those affected. Its biological and clinical complexity makes ALS one of the major challenges in contemporary biomedical research, driving the development of new tools capable of providing a more precise understanding of the molecular mechanisms underlying its onset and progression.
Although there is still no cure, advances in genetics, molecular biology and omics technologies have significantly transformed our understanding of neurodegenerative diseases in recent decades. Today, it is known that processes such as altered gene expression, protein aggregation, loss of cellular homeostasis and changes in neuronal function play a key role in their development. However, translating this knowledge into diagnostic and therapeutic solutions still requires increasingly sophisticated biological tools and highly interdisciplinary approaches.
In this context, CIC bioGUNE, a member of BRTA, conducts research aimed at understanding the fundamental mechanisms of life and generating new technologies to drive the next generation of biomedical applications. The combination of synthetic biology, genomics, structural biology, bioengineering and data analysis is making it possible to explore innovative approaches to the study of complex diseases and to move towards more precise and personalised medicine.
Exploring evolution to design new biological tools
Among the centre’s most innovative lines of research is the work carried out by the Synthetic Biology Lab, led by Ikerbasque Prof. Raúl Pérez-Jiménez, which investigates how molecular evolution can become a source of new tools for biomedicine.
Using ancestral protein reconstruction techniques, the team studies molecules that existed millions of years ago in order to identify biological properties that are no longer found in present-day organisms. This approach has enabled the development of new variants of CRISPR-Cas systems with distinctive features for gene editing, expanding the possibilities for accessing and modifying genetic material.
Alongside this line of research, the laboratory investigates new strategies based on enzyme engineering, mechanobiology and advanced biomaterials. In addition, a research line led by Dr Ylenia Jabalera, Ikerbasque researcher and La Caixa Junior Leader at CIC bioGUNE, is developing new gene-editing tools inspired by natural mechanisms capable of inserting DNA in a targeted manner. These technologies could open up new opportunities to address diseases such as ALS, where the diversity of mutations makes the development of treatments particularly challenging. This line of research has been supported by FUNDELA and, although it is fundamental research, all these efforts share a common goal: to generate knowledge and tools that allow us to better understand the biological processes involved in health and disease.
Understanding biological complexity to advance precision medicine
Neurodegenerative diseases, including ALS, highlight the need to approach biology from a multidisciplinary perspective. The identification of genetic factors associated with the disease, such as certain variants in genes involved in neuronal function, has shown that understanding the molecular origin of these conditions is an essential step towards developing more specific and effective treatments.
The growing capacity to analyse large volumes of genetic and molecular information is enabling progress towards precision medicine models capable of adapting diagnoses and therapies to the biological characteristics of each patient. This paradigm shift requires new experimental tools, new analytical capabilities and robust basic research that makes it possible to interpret the complexity of biological systems.
For this reason, much of the research carried out at CIC bioGUNE not only seeks to answer fundamental scientific questions, but also to lay the technological foundations that will make it possible to address, in the future, diseases for which significant unmet medical needs still exist.
Research as an investment in the future
World ALS Day is also an opportunity to reflect on the role research plays in building a society that is better prepared to face the health challenges of the future. Many of the advances that are beginning to transform medicine today originate in basic research which, at its outset, sought to understand how the most fundamental mechanisms of life work.
The ability to reconstruct ancestral proteins, design new gene-editing tools or decipher the molecular complexity of a neurodegenerative disease are examples of how science constantly expands the boundaries of knowledge. Turning this knowledge into real solutions for patients requires time, collaboration and a sustained commitment to research excellence.
At CIC bioGUNE, the commitment to knowledge generation, technological innovation and interdisciplinary research continues to drive the development of key scientific capabilities to address some of the major biomedical challenges of the present and the future.
.png)
