UCL Great Ormond Street Institute of Child Health


Great Ormond Street Institute of Child Health


Prof Stephen Hart

Professor in Molcular Genetics

Genetics & Genomic Medicine Dept

UCL GOS Institute of Child Health

Joined UCL
1st Apr 2002

Research summary

Gene therapy offers exciting opportunities for the development oftherapies in virtually all areas of medicine. Recent clinical advances arestarting to realise this potential although there remain many challenges amongst which delivery is a key issue. Much of my research has thereforefocused on this challenging area to develop delivery methods compatible withclinical use. In particular I have developed a family of synthetic gene delivery formulations, or nanoparticles, from conceptual stages through to treatment of disease models in vivo.  More recently we have extended our formulations to the delivery of siRNA and other oligonucleotides as well as imaging contrast agents and radiolabels for real-time imaging of delivery in vivo.

 I have established a number of collaborations, exploiting the breadth of high quality research groups at UCL, including Departments of Chemistry, Biochemical Engineering, Centre for Respiratory Medicine, and the Centre for Cardiovascular Biology and Medicine. These collaborations have led to highly productive interdisciplinary hypothesis-driven research, involving synthesis ofnovel reagents, studies to establish the fundamental rules and relationshipsconcerning vector structure and function and correction of animal models ofdisease.

Overall the research areas are non-viral vector development and gene therapy for: 1) gene therapy for cardiovascular diseases (bypass grafts), 2) gene therapy for respiratory diseases (cystic fibrosis), 3) gene therapy for cancer (neuroblastoma), 4) gene therapy for the brain, 5) further vector development.

Teaching summary

My teaching activity mainly concerns training of research students from MSc to PhD levels.


University of Cape Town
Doctorate, Doctor of Philosophy | 1991
University of the Witwatersrand
Other higher degree, Master of Science | 1986
University of Liverpool
First Degree, Bachelor of Science (Honours) | 1981