Introduction
RNA therapies for both rare and common diseases (cardiovascular and neurodegenerative diseases, cancer, etc.) have witnessed a powerful acceleration with the success of COVID-19 mRNA vaccines and also with the approval of eleven oligonucleotide-based drugs in recent years. Therefore, these nucleic acids-based treatments are gaining increasing social, political, and economic interest.
This program aims to train students in all relevant aspects of the design and preclinical and clinical testing of RNA therapies, acquiring the overall knowledge of the challenges associated with their development, their delivery, and the regulatory steps needed to get them to the clinics and the market, as well as learning how to effectively and adequately communicate among stakeholders (to patients, families, caregivers, healthcare professionals, science communicators, and the lay public).
Main Topics Addressed
Students will learn theoretical and practical aspects of:
- nucleic acid chemistry and types of RNA drugs;
- design of RNA drugs depending on the specific application needed;
- in vitro and in vivo models for preclinical testing;
- safety and toxicology issues;
- regulatory issues;
- knowledge and technology transfer;
- communication to relevant stakeholders.
Students will be able to revise scientific literature to extract relevant information about a disease or the technical application of RNA therapies, to analyse the restuls and identify the limitations of the research, and to have the ability to communicate the results in different environments (scientific and non-scientific).
Upon completion, students will be competent in interdisciplinary and transdisciplinary views and researcha pproaches contributing to the development of RNA therapies.
On the practical side, they will be competent in identifying diseases/genes/variants that can be targeted and the specific desing of the therapeutic approach. They will be competent to use scientific data to reach a conclusion, understand the challenges and limitations of the research and have the ability to communicate the results to non-scientific people.
Skills include the ability to identify one’s capacities, deal with complexity, critically reflect, learn and work both collaboratively and autonomously and to communicate constructively in different environments, understanding different viewpoints, as well as the ability to create confidence and feel empathy. This course is specially usefull for upskilling and reskilling given the relevance and novelty of the topic in biomedical careers.
Practical informations
Study domain: Natural Sciences, mathematics and statistics / Health and Welfare
Related HUB: Hub 3 Health
Target audience: PhD, Lifelong Learners; Researchers; Laboratory Technicians
Language:English
ECTS: 75h, 3 ECTS
Assessment
Students are required to complete a number of tasks and participation and assistance of the students to different
activities will also be considered.
Assesment methods include the following:
- short tests after lectures, evaluating the acquisition of theoretical aspects and the practical ability to identify and design specific applications of RNA therapies
- online oral powerpoint presentation and summary for lay public based on a given article, evaluating the student's ability to extract relevant information from scientific literature and to communicate it to their peers and to non-scientific people
- written short report of a webinar of choice, evaluating their capacity to understand the objectives and limitations of specific research topics and to summarise the results.
Application process
PhD students working in related topics will be prioritised.
Curriculum prerequisites: Degree in biochemistry, biotechnology, biology, biomedicine, chemistry, chemical engineering, pharmacy
or related.
Competences prerequisites: knowledge in human molecular genetics and in
molecular biology techniques
Contact: Lourdes Ruiz: lourdes.ruiz@uam.es
Professors
- Thorsten Schmidt – Professor of Molecular Medicine, Eberhard Karls University of Tübingen
- Faiza Fakhfakh – Professor of Molecular and Human Genetics and Head of the Molecular and Functional Genetics Laboratory (LGMF), University of Sfax
- Sorina Dinescu – Associate Professor, Department of Biochemistry and Molecular Biology, Faculty of Biology, University of Bucharest
- Cecilia Battistelli – Associate Professor, Department of Molecular Medicine, Sapienza University of Rome
Expert lecturers for webinars:
Non-CIVIS Universities: Prof. A. Aartsma-Rus, University of Leiden, Netherlands; Prof. M.A. Denti, University of
Trento, Italy; Alex Garanto, Radboud University, Netherlands.
Research Institutions: Prof. V. Arechavala-Gomeza, Biobizkaia Health Research Instiute, Spain
Pharma/Biotech companies: B. Llamusi, Arthex Biotech, Spain
Parents' assocations: M. Montolio, Duchenne Parents Project Spain
Science dissemination expert: Prof. L. Montoliu (Center for Biotechnology-CSIC, Madrid, Spain)
Prof. A. Aartsma-Rus, Prof. of Translational Genetics, Leiden University Medical Center, University of Leiden
Prof. M.A. Denti, Associate Professor, University of Trento
Prof. A. Garanto, Associate Professor, Radboud University Medical Center
Prof. V. Arechavala-Gomeza, Ikerbasque Research Professor, Biobizkaia Health Research Insitute
B. Llamusi, PhD, CEO and CO-founder, Arthex Biotech
M. Montolio, PhD, Scientific Director Duchenne Parents Project Spain
Prof. L. Montoliu, research professor Spanish National Research Council (CSIC), with ample experience in social
media for science dissemination (animal experimentation, rare diseases, ethics...) Won several prestigious awards
for sceince dissemination activities