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Summary

The aim of this module is to teach you the historic, current and future cell and gene therapy treatment strategies. It will introduce concepts in clinical and basic research, clinical trials and methodology. In particular it will cover: Principles of stem cell transplantation, including bone marrow transplantation; rationale for treating particular diseases by cell and gene therapy; principles and practice of clinical trials of cell therapy (e.g. clinical trials for genetic blood disorders, clinical trials for muscle, liver, skin, lung and metabolic disorders), including stem cell transplantation, immunotherapy and gene therapy; discussion of regulation and monitoring required for such clinical studies.

Learning Objectives and Outcomes

After taking this module, you should be able to:

1. Understand the process, principles and regulation of bringing cell and/or gene therapies from the bench to the bedside
2. Have awareness of the current scope of cell and gene therapy in clinical practice
3. Have a grounding in cell and gene therapy technologies sufficient to form a generalised plan for moving a novel laboratory finding to clinical practice

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 ICH/¹û¶³Ó°Ôº staff with some external experts. There will be the opportunity for you to attend additional ICH/¹û¶³Ó°Ôº research seminars, journal clubs and lab meetings (though this is not compulsory).

Selected Reading List

Sadelain, M., Rivière, I. & Riddell, S. Therapeutic T cell engineering. Nature 545, 423–431 (2017).Ìý

Critchley B, Gaspar HB, Bendetti S. (2022) Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.Ìý Mol Ther doi: 10.1016/j.ymthe.2022.11.015Ìý

Cantore A, Cantore A, Fraldi A, Meneghini V, Gritti A (2022) In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges Front Med doi: 0.3389/fmed.2021.774618Ìý

Cavazzana M et al., (2019) Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges. Nature reviews Drug Discovery 18:447-462Ìý