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Title Nucleic Acid Chemistry and Therapy
Course number 3190
Programme Utveckling och regeneration (DevReg)
Language English
Credits 3.0
Date 2023-05-08 -- 2023-05-29
Responsible KI department Institutionen för biovetenskaper och näringslära
Specific entry requirements
Purpose of the course The purpose of the course is to provide participants with good and up to date knowledge of nucleic acid chemistry and oligonucleotide therapy. The intention is to increase general knowledge about nucleic acids and how these can undergo different reactions as well as how oligonucleotides are chemically synthesized and modified for therapeutic or other use. We expect that this will stimulate and inspire the students in their own research whether they work on nucleic acid biology, therapy, analyse nucleic acids or use oligonucleotides as tools for investigations. It is an intention to increase understanding of nucleic acids, how these molecules work and how they can be used in therapy. In addition, knowledge on how oligonucleotides are synthesized and how these can be modified will enhance the insight and enable the students to improve their use of oligonucleotides as tools or potential therapeutics.
Intended learning outcomes At the end of the course the students
- should be able to explain the underlying chemistry of nucleic acids and how these can react at different parts of the structure.
- should be able to explain how oligonucleotides can be synthesized and modified and why currently used modifications and conjugations are done.
- should be able to make selections of modifications and to design oligonucleotides, for use as potential therapeutics.
Contents of the course Introduction to Nucleic acid chemistry and nomenclature
Reactions at nucleic acid bases
Reactions at ribose and deoxyribose sugars
Reactions at and nomenclature of phosphates and phosphate modifications
Methods for synthesis of native and modified oligonucleotides
Common modifications used for oligonucleotide therapy
Introduction to Oligonucleotide therapy
Antisense, pre-mRNA Splice-switching, siRNA, mRNA and DNA-targeting ON therapy
The problem of oligonucleotide delivery in therapy
Teaching and learning activities The course consist of lectures by experts and seminars in the presence of experts on different aspects of nucleic acid chemistry and oligonucleotide therapy as well as workshop activity including presentations by the students. Additional home work combined with individual and group activities in the class room will be a part of the learning.
Compulsory elements The lectures, seminars and workshop activities with student presentations will be compulsory. Absence will be compensated by extra assignments. The student will also submit reports from a workshop in written form for review and approval, in connection to the student presentations.
Examination Oral presentations on workshops as well as a written account with specific course related questions.
Literature and other teaching material Recommended books:
- Nucleic Acids in Chemistry and Biology. Editors: G M Blackburn, M J Gait, D Loakes, D M Williams. RSC Publishing
- Advanced Organic Chemistry of Nucleic acids. Z. Shabarova, A Bogdanov, VCH Verlagsgesellschaft.
In addition a couple of the most up to date review articles will be recommended to registered students ahead of the course start.
Number of students 8 - 16
Selection of students Selection will be based on 1) the relevance of the course syllabus for the applicant's doctoral project (according to written motivation), 2) start date of doctoral studies (priority given to earlier start date)
More information Schedule to be constructed, but Ascension day (18 May) and the day after will be free from teaching and the last day will only be for returning tasks.
Additional course leader
Latest course evaluation Course evaluation report
Course responsible Roger Strömberg
Institutionen för laboratoriemedicin
0852481024
roger.stromberg@ki.se

Neo, Hälsovägen 9

14183
Huddinge
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