Generative AI is more efficient than nature at designing proteins to edit the genome
After studying the diversity of mobile elements in the genome to feed and train generative AI tools, researchers have been able to design and lab validate new synthetic proteins that can edit the genome more efficiently than natural proteins.
2025-10-02
(Press-News.org)
Researchers at Integra Therapeutics, in collaboration with the Pompeu Fabra University (UPF) Department of Medicine and Life Sciences (MELIS) and the Center for Genomic Regulation (CRG), have designed and experimentally validated new synthetic proteins that can edit the human genome more efficiently than proteins provided by nature. This work, a global pioneer published today in the journal Nature Biotechnology, will be of great use in improving the current gene editing tools used in biotechnology research and personalized medicine by developing cellular (CAR-T) and gene therapies, especially to treat cancer and rare diseases.
The ability to insert large DNA sequences into genomes in a safe, targeted manner has been a revolution in the research and development of advanced therapies in recent years. Among the most promising systems are transposases, such as PiggyBac, which copies and pastes DNA to introduce therapeutic genes into patient cells. However, their potential has been limited by the scarce diversity of known transposases and their lack of precision.
Exploring biodiversity
The researchers used computational bioprospecting to screen more than 31,000 eukaryotic genomes and discovered more than 13,000 new, previously unknown PiggyBac sequences. After performing experimental validation in cultured human cells, 10 active transposases were identified, demonstrating that there is a large functional diversity that has not yet been explored. Two of these new transposases showed activity comparable to versions already optimized for laboratory and patient use, and one of them even exhibited high activity in human primary T cells, a crucial cell type for cancer therapies.
Designing with generative artificial intelligence
In the second phase, researchers went beyond nature and used a protein large language model (pLLM), a form of generative artificial intelligence. They trained the model with the 13,000 PiggyBac sequences discovered to generate completely new sequences with enhanced activity. This approach not only optimized one of the existing transposases, but also demonstrated that AI-engineered variants are compatible with advanced gene editing technologies such as the FiCAT platform.
“Publishing this paper in Nature Biotechnology opens the way to revolutionizing the field of gene editing and advanced therapies and cements Integra Therapeutics’ position at the forefront of gene therapies and the use of innovative tools like AI for protein design in our development,” notes Dr. Avencia Sánchez-Mejías, CEO and co-founder of Integra Therapeutics.
“For the first time, we have used generative AI to create synthetic parts and expand nature. Like the cognitive power of ChatGPT can be used to write a poem, we have used the protein-based large language models to generate new elements that comply with the physical and chemical principles of genes,” explains Dr. Marc Güell, scientific director at Integra Therapeutics and ICREA researcher at MELIS-UPF where he heads up the Translational Synthetic Biology Lab.
“These AI models are trained with all known protein sequences on earth and learn the internal language or ‘grammar’ of proteins. Using this grammar, they are able to speak this language perfectly, generating completely new proteins that maintain structural and functional meaning,” says Dr. Noelia Ferruz, who leads the Artificial Intelligence for Protein Design Group at the CRG.
To accelerate and expand its FiCAT technology and pipeline of therapeutic products, Integra Therapeutics forges strategic partnerships with leading companies and research centers like UPF and CRG.
END
ELSE PRESS RELEASES FROM THIS DATE:
2025-10-02
Firms in the Gulf region are increasingly expected to align with environmental, social, and governance (ESG) standards. Yet the link between ESG performance and financial outcomes remains ambiguous. A new study examines whether publicly listed companies in GCC member states that achieve high ESG ratings also deliver superior financial performance.
The study “ESG and financial performance in the Gulf Cooperation Council” was authored by Catalina Stefanescu-Cuntze, professor of management science and faculty lead of the Master in Analytics and Artificial Intelligence Program at ESMT Berlin, together with Rodrigo Tavares and Catarina Sá from Nova School ...
2025-10-02
University of Michigan researchers have developed a tool powered by artificial intelligence that can help them examine the behavior of a single molecule out of a sea of information in the blink of an eye—or at least overnight.
Understanding the behavior of single molecules is important: it can lead to knowledge of different cellular processes or track how diseases begin and progress. To track the behavior of single molecules, researchers tag the molecules with what's called a fluorophore. They excite these fluorophores with a laser, then use powerful microscopes to follow the behavior of the tagged molecules over time.
But ...
2025-10-02
Western cultures tend to view teens as moody, irresponsible, and rebellious. However, in China and in other cultures, the teen years are viewed more positively and seen as a time of learning how to become responsible and fulfilling family obligations. Previous research shows that fulfilling family obligations is an important marker of maturity and that as adolescents continue to improve their skills, they can contribute more to their families.
Past studies also show that when teens are seen ...
2025-10-02
A groundbreaking study published today in the European Geosciences Union (EGU) journal Earth System Dynamics provides a critical and previously underestimated connection between Antarctic sea ice, cloud cover, and global warming. This research is important because it shows that a greater extent of Antarctic sea ice today, compared to climate model predictions, means we can expect more significant global warming in the coming decades.
The study, led by Linus Vogt from Sorbonne University, utilized an emergent constraint based on data from 28 Earth system models and satellite observations ...
2025-10-02
Researchers have found the first direct evidence that tiny particles of air pollution stick to our red blood cells, meaning they can travel freely around the body.
These particles are produced by vehicle exhausts and from brake and tyre wear, and can easily enter the lungs. They have recently been found in the brain and heart, where they are linked to increased risk of disease. However, until now, there has been no conclusive evidence of how pollution particles infiltrate these parts of the body.
In the ...
2025-10-02
As most of us have experienced, when multiple people work together, things sometimes turn out better than doing the same task alone. Then, there are the other times, when cooperation actually seems to hinder progress, making things much more inefficient.
To understand why this ‘cooperative advantage’ manifests in some cases but not in others, a research group led by Assistant Professor Asuka Takai of the Graduate School of Engineering at Osaka Metropolitan University conducted a task where participants used a robotic arm ...
2025-10-02
The University of Gothenburg is establishing the Crown Princess Victoria Professorship in Biodiversity. The professorship is a gift ahead of the Crown Princess's 50th birthday in 2027, in collaboration between Carl Bennet AB and the University of Gothenburg.
Biodiversity is a growing field of research, and the University of Gothenburg now has the opportunity to contribute more effectively through a new professorship in biodiversity established in the Crown Princess's name. The Crown Princess has repeatedly expressed her belief that the diversity ...
2025-10-02
A groundbreaking technology developed by Heriot-Watt University has received over £475,000 in funding to revolutionise how eczema is diagnosed and treated, potentially ending years of suffering for millions of patients across the UK.
The innovative vibroacoustic sensor, which measures material changes in each layer of skin using small vibrations on the surface, has received £275,000 from Scottish Enterprise and just over £200,000 from the Medical Research Council's Gap Fund to advance clinical ...
2025-10-01
Commercial sunbeds should be banned in the UK, argue experts in The BMJ today.
Using sunbeds causes melanoma and other skin cancers, particularly among young people, yet existing sunbed legislation is ineffective and there is little evidence that stricter rules would help protect the most vulnerable, say Professor Paul Lorigan and colleagues.
Indoor tanning is experiencing a boom in popularity, particularly among Gen Z (born 1997-2012), with social media promoting sunbeds as integral to wellness, they explain. For example, a 2024 survey of 2,003 people in the UK by Melanoma Focus ...
2025-10-01
The healthcare community has an important role in opposing the death penalty, argues an expert in The BMJ today.
Bharat Malkani, reader in law at Cardiff University, says doctors must refuse to participate in the execution process and speak out against a practice that is antithetical to their commitment to promote health and wellbeing.
Amnesty International reports that although the number of countries carrying out executions is decreasing, the number of executions in countries where it is legal has increased in recent years, he explains.
At least 1518 executions took place globally in 2024, rising from 1153 recorded in 2023, making it the fourth ...
LAST 30 PRESS RELEASES:
[Press-News.org] Generative AI is more efficient than nature at designing proteins to edit the genome
After studying the diversity of mobile elements in the genome to feed and train generative AI tools, researchers have been able to design and lab validate new synthetic proteins that can edit the genome more efficiently than natural proteins.
