During this course, you will cover the most up-to-date developments on gene and nucleic acid-based therapies. This will include a review of pertinent pre-clinical and clinical research related to cystic fibrosis, haemophilia, SCID and other diseases for which gene therapy and genome-based therapies have advanced over the last 10 years.
Learn from leading experts in genomics about:
- Basic tools related to gene and nucleic acid-based therapies, such as commonly used viral and non-viral vectors and vector production methods.
- The use of anti-sense technology, genome editing and applications involving small non-coding RNAs.
- How the identification and characterisation of mutations and polymorphisms have led to development of personalised medicines and mutation-specific treatments for a range of diseases.
- Basic concepts related to intellectual property, commercialisation strategies and health economics.
By the end of the module you will be able to:
- Explain how genetic information contributes to drug development and the concepts in developing gene and nucleic acid-based therapies from genomic studies.