Cell and Gene Therapy Course Descriptor

Course Summary

This course introduces and explores the principles, theory and concepts that underpin cell and gene therapy. This rapidly advancing area in bioscience is recognised as having the potential to revolutionise therapy for inherited disorders and many other diseases. The course starts by examining the implications of the human genome project and reviews the function of DNA, chromosomes, RNA transcription and translation. It then considers the significance of Recombinant DNA techniques, and the use of mRNA and vector design in effective gene therapy. The course also considers the impact of cell therapy and the characteristics and significance of stem cells  and the issues and challenges faced in transplantation and integration, alongside ethical and regulatory concerns.

Course Aims

• To provide learners with the principles, theory and concepts that define and distinguish cell and gene therapy and the wider scientific and cultural contexts in which they are being developed, applied and the associated ethical and regulatory issues.

• To provide learners with detailed insight into the challenges and potential of cell and gene therapy to address inherited disorders and disease.

• Train learners to engage systematically with the methodologies, techniques, tools and processes that enable cell and gene therapy.

Learning Outcomes

On successful completion of the course, learners will be able to:

Knowledge and Understanding

Subject Specific Skills

Transferable and Professional Skills

Teaching and Learning

This is an e-learning course, taught throughout the year. 

This course can be offered as a standalone short course.

Teaching and learning strategies for this course will include: 

• Online learning
• Online discussion groups
• Online assessment

Course information and supplementary materials will be available on the University’s Virtual Learning Environment (VLE).

Learners are required to attend and participate in all the formal and timetabled sessions for this course. Learners are also expected to manage their self-directed learning and independent study in support of the course.

The course learning and teaching hours will be structured as follows:

• Off-the-job learning and teaching (6 days x 7 hours) = 42 hours
• One-the-job learning (12 days x 7 hours) = 84 hours (e.g. 2 days per week for 6 weeks)
• Private study (4 hours per week) = 24 hours

Total = 150 hours

Workplace assignments (see below) will be completed as part of on-the-job learning.

Assessment

Formative

Learners will be formatively assessed during the course by means of set assignments. These will not count towards the final degree but will provide learners with developmental feedback. 

Summative

Assessment will be in two forms:

Feedbacks

Learners will receive formal feedback in a variety of ways: written (via email or VLE correspondence) and indirectly through online discussion groups. Learners will also attend a formal meeting with their Academic Mentor (and for apprentices, including their Line Manager). These bi or tri-partite reviews will monitor and evaluate the learner’s progress.  

Feedback is provided on summatively assessed assignments and through generic internal examiners’ reports, both of which are posted on the VLE.

Indicative Reading

Note: Comprehensive and current reading lists for courses are produced annually in the Course Syllabus or other documentation provided to learners; the indicative reading list provided below is used as part of the approval/modification process only.

Books 

• Watson, J. D. (1965). Molecular biology of the gene. New York : W. A. Benjamin.
• Vertès, A., Dowden, N. J., Smith, D. and Qureshi, N. (2020). Second Generation Cell and Gene-based Therapies : Biological Advances, Clinical Outcomes and Strategies for Capitalisation. London ; San Diego, CA : Academic Press
• Turnpenny, P. D., Ellard, Sian, & Cleaver, Ruth. (2021). Emery’s Elements of Medical Genetics and Genomics (Sixteenth edition.). Philadelphia, PA : Elsevier 2021

Journals

Learners are encouraged to read material from relevant journals on cell and gene therapy as directed by their course leader.

Electronics Resources

Learners are encouraged to consult relevant websites on cell and gene therapy.

Indicative Topics

• Cell structure and function
• Modern and experimental therapeutics
• Disease