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Summary

The aim of this module is to teach students the basic scientific concepts which underlie cell and gene therapy. The module will introduce aspects of genetics, cell biology, virology, and immunology. It will cover: basic molecular concepts, viral vector technologies, non-viral vector technologies, genome editing, safety issues in cell and gene therapy, new concepts in cell therapy and preclinical applications in cell and gene therapy.Ìý

Learning Objectives and Outcomes

After taking this module, you should be able to:

1. Gain a critical understanding of the basic scientific concepts underlying cell and gene therapy.
2. Understand the design of different vectors and the advantages and disadvantages of non-viral and different viral delivery techniques.
3. Identify potential pre-clinical applications and challenges of the different cell and gene therapy approaches.

Who is this module for?

This is a core module for the MSc Cell and Gene Therapy.

Teaching and Learning Methods

This module will be delivered mostly as a series of lectures by GOS ICH/Ïã¸ÛÁùºÏ²Ê staff with some external experts. There will be the opportunity for you to attend additional GOS ICH/Ïã¸ÛÁùºÏ²Ê research seminars.

You are expected to invest significant time to self-study outside of class meetings.

Assessment

This module will be assessed by a 1.5 hour unseen exam with multiple choice questions.

Selected Reading List

Anzalone, A.V., Koblan, L.W. & Liu, D.R. Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors. Nat Biotechnol 38, 824–844 (2020). Ìý

Bashor, C.J., Hilton, I.B., Bandukwala, H. et al. Engineering the next generation of cell-based therapeutics. Nat Rev Drug Discov 21, 655–675 (2022). Ìý

Cring, M.R., Sheffield, V.C. Gene therapy and gene correction: targets, progress, and challenges for treating human diseases. Gene Ther 29, 3–12 (2022). Ìý

Kimbrel, E.A., Lanza, R. Next-generation stem cells — ushering in a new era of cell-based therapies. Nat Rev Drug Discov 19, 463–479 (2020). Ìý