Module Overview

Gene Therapy and Genetic Engineering

This module introduces the student to eukaryotic gene structure, gene expression and regulation. It describes the role of DNA binding proteins in gene regulation, how transcription is initiated in eukaryotic organisms and controlled through chromatin structure, activators, repressors, mediators and chromatin modifying enzymes. The structure, function and regulation of the spliceosome will be discussed alongside examples of alternative and aberrant splicing.

The module also aims to give the student a comprehensive review of genetic engineering technologies and applications in research, medicine and industry. This module aims to provide understanding of the principles, technologies and risks involved in gene therapy and to evaluate ethical debate surrounding gene editing and gene therapy approaches.

Module Code

BIOL 3090

ECTS Credits


*Curricular information is subject to change

Lecture Content

  • Eukaryotic gene structure and gene expression.
  • Initiation and regulation of eukaryotic transcription; RNAP I, II and III.
  • DNA binding proteins – HTH, zinc fingers, leucine zippers.
  • Chromatin structure and influence on gene regulation; activators, repressors, mediators, chromatin modifying enzymes.
  • DNA methylation and regulation of gene expression.
  • Spliceosome structure and function, alternative/aberrant splicing, SR proteins.
  • Introduction to Genetic Engineering and Gene Therapy – technologies, application to disease and ethics.
  • Molecular techniques e.g. plasmids, vectors, cloning, PCR, real-time PCR.
  • Molecular Profiling e.g. DNA fingerprinting/profiling/testing, RFLP, PCR.
  • Functional genomics and the use of model organisms.
  • Viral, non-viral and gene correction approaches to gene therapy.
  • Genome Editing and application to treat human disease e.g. ZFNs, TALENS, CRISPR.

Practical Content

  • Bioinformatics – sequence acquisition, PCR primer design, primer ordering.
  • DNA, RNA and protein isolation, quantitation and qualification.
  • Optimisation of PCR conditions.
  • Diagnostic PCR and RT-PCR  in genetic disorders.
  • Transfection of mammalian cells in culture and analysis of gene expression using quantitative and in-situ assays.

Lectures, tutorials, online student response systems, online movie animations, in-class quizzes, scientific paper review and laboratory-based practical sessions.

Module Content & Assessment
Assessment Breakdown %
Formal Examination60
Other Assessment(s)40