Welcome to RNA Medicine – From Bench to Bedside
Sakari Kauppinen, Professor and Director, Center for RNA Medicine
Mogens Vyberg, Professor of Clinical Pathology, Center for RNA Medicine
Marianne Løvendorf, Senior Postdoc, Center for RNA Medicine
Anja Holm, Senior Postdoc, Center for RNA Medicine
Kasper Thystrup, PhD Student, Center for RNA Medicine
ECTS: 5 ECTS points
Time: November 2-6, 2020
Place: Center for RNA Medicine, Dept of Clinical Medicine, Frederikskaj 10B, AAU Campus Copenhagen
Zip code: DK-2450 Cph SV
Number of seats: 16
Deadline: October 1, 2020
Description: One of modern biology’s biggest surprises was the finding that the human genome contains only about 21.000 protein-coding genes, comprising less than 2% of the genome sequence. However, recent data imply that the human genome is pervasively transcribed and encodes tens of thousands of non-protein coding RNAs (ncRNAs) that play critical regulatory roles in numerous biological processes. An improved understanding of basic RNA biology and the complexity of the human transcriptome has triggered a corresponding revolution in development of RNA-based therapies, which hold promise for the treatment of a wide range of life-threatening diseases. This is highlighted by the recent FDA approvals of three RNA medicines: (i) the antisense oligonucleotide (ASO)-based drugs eteplirsen for treatment of Duchenne muscular dystrophy and (ii) nusinersen for treatment of the neurodegenerative disease spinal muscular atrophy, respectively, and (iii) the siRNA drug patisiran for the treatment of hereditary transthyretin amyloidosis with polyneuropathy. Notably, RNA drugs can be designed to target essentially any RNA transcript encoded by the genome and can thus target a large part of the currently undruggable genome.
This PhD course will provide the participants with an overview of ncRNA biology and development of RNA-targeted therapeutics, and consists of a series of research seminars and laboratory exercises. The seminars will cover key topics in the rapidly expanding field of RNA research from ncRNA biology, high-throughput RNA sequencing (RNAseq) methods and computational analysis of RNAseq data to the emerging roles of ncRNAs in human disease and development of RNA-based medicines. Each seminar will be followed by a scientific discussion of the participating PhD students with the lecturer. As preparation, two scientific papers selected by the lecturer have to be read prior to the PhD course. The laboratory exercises are designed to provide hands-on experience with key methods and tools used in ncRNA research and discovery of RNA-targeted therapeutics.
- Khvorova A & Watts JK. The chemical evolution of oligonucleotide therapies of clinical utility. Nat. Biotechnol. 35: 238–248 (2017).
- Lim KR, Maruyama R & Yokota T. Eteplirsen in the treatment of Duchenne muscular dystrophy.
Drug Des Devel Ther. 11: 533-545 (2017).
- Corey, DR. 2017. Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy. Nat Neurosci. 20: 497–499 (2017).
- Setten RL, Rossi JJ & Han SP. The current state and future directions of RNAi-based therapeutics. Nat Rev Drug Discov. 18: 421-446 (2019).
Prerequisites: PhD students in biomedical research or molecular biology with an interest in ncRNA biology in human health and disease and discovery and development of RNA-targeted therapeutics should apply.
Evaluation: Participants who have engaged actively in all parts of the course and completed all lab exercises satisfactorily will be awarded a certificate of completion at the end of the PhD course. The workload corresponds to 5 ECTS points, and includes one week of preparation prior to the course (2 articles for each lecture that should be read in advance).
Important information concerning PhD courses: We have over some time experienced problems with no-show for both project and general courses. It has now reached a point where we are forced to take action. Therefore, the Doctoral School has decided to introduce a no-show fee of DKK 3,000 for each course where the student does not show up. Cancellations are accepted no later than 2 weeks before start of the course. Registered illness is of course an acceptable reason for not showing up on those days. Furthermore, all courses open for registration approximately four months before start. This can hopefully also provide new students a chance to register for courses during the year. We look forward to your registrations.
- Teacher: Sakari Kauppinen