790053 Non-viral gene therapy

Type
Lecture and seminar
Semester hours
3
Lecturer (assistant)
Feichtinger, Georg
Organisation
Offered in
Sommersemester 2024
Languages of instruction
Englisch
Content

Module content:
- Definition and differentiation from other biological therapies
- Classification of gene therapies (non-viral vs. viral, transient vs. stable, somatic vs. germline, episomal vs. integrating, ex vivo vs. in vivo, local vs. systemic)
- Classification of gene therapeutic interventions (replacement/augmentation, genome editing, splicing modulation, ASO and RNAi knochdown, CRISPRa, CRISPRi)
- Overview of nucleic acid payloads employed in non-viral gene therapy (ASOs, siRNAs, mRNA, oRNA, saRNA, pDNA, dbDNA/MIDGE)
- Overview of common drug delivery methods for non-viral gene therapy (lipid nanoparticles, liposomes/lipoplexes, nanoparticles, conjugates) including specific requirements (efficacy, safety, targeting)
- Overview of biopharmaceutical production/CMC for non-viral gene therapies (API manufacturing and formulation: Upstream, downstream, QC)
- Introduction to fundamental regulatory considerations with relevance to non-viral gene therapies (FDA/EMA aligned focus on particular aspects of non-viral gene therapies: preclinical, IND-enabling and early clinical phase considerations)
- Group assignments and presentations regarding examples of different types of non-viral gene therapies for a variety of therapeutic indications using selected (literature) examples
- Discussion of ethical implications and aspects of science communication, education and patient involvement with regards to establishing informed acceptance of novel therapies and their transformative potential for modern medicine within the wider society
Previous knowledge expected

Fundamental understanding of genetics, biochemistry, and cell biology. Solid foundation in molecular biology.
Objective (expected results of study and acquired competences)

After the completion of this module students will be able to lead informed and discerning dialogue and discussions with colleagues and laymen to impart a fundamental understanding of gene therapies.
You can find more details like the schedule or information about exams on the course-page in BOKUonline.