Synthetic Biology in Gene Therapy and Vaccines

Learning Outcomes

• Explain at an advanced level how synthetic biology tools can be applied in gene therapy strategies.
• Understand and critically evaluate modern editing and engineering technologies of the immune system
• Design and implement strategies for constructing synthetic genetic circuits, metabolic networks, and engineered biological systems.
• Apply the Design-Build-Test-Learn (DBTL) framework to the development and optimization of synthetic biological systems

Module Syllabus

Session 1 – Foundations and Development of Gene Therapies

Introducing the concept and early history. Real case: First gene therapy attempt, discussion about immune reaction to adenoviral vectors and the importance of safety engineering. Case studies: a) gene therapy targeting the eye (blindness treatment), b) spinal muscular atrophy (Viral vector engineering) and c) mRNA delivery (non-viral delivery)

Session 2 – CAR T cell therapies

Introducing concepts; synthetic receptors, immune cell engineering and logic-based targeting. How CAR-T cells are designed and used. Case reports and future directions

Session 3 – Bridging gene therapies with next generation vaccines

History of vaccinations, national schemes and target populations. Use of synthetic biology in vaccine development and future directions. How ethics are involved in these approaches.

Suggested Bibliography

• Gene and Cell Therapies for Beta-Globinopathies, Editors:Punam Malik, John Tisdale, Copyright: 2017
• Fast Facts: CAR T-Cell Therapy: Insight into current and future applications (Free), By Richard J. Buka; Ankit J. Kansagra, S.Karger AG DOI: https://doi.org/10.1159/isbn.978-3-318-06742-2, Status: Available ISBN electronic: 978-3-318-06742-2
• Vaccines and Immunizations-World Health Organization-(https://www.who.int/health-topics/vaccines-and-immunization#tab=tab_1)