Prodessor Stephen HArt
PROFESSOR IN MOLECULAR GENETICS, UCL GREAT ORMOND STREET INSTITUTE OF CHILD HEALTH
Samantha Renke is an award-winning actress and disability campaigner.
Stephen Hart is a professor in molecular genetics at UCL Great Ormond Street Institute of Child Health and deputy head of the Genetics and Genomic Medicine Programme. He obtained his PhD from the University of Cape Town and has worked in the field of genetic therapies for more than 20 years, with more than 100 publications. His current research activities include the development of synthetic nanoparticles for the delivery of nucleic acid therapeutics including siRNA and mRNA for the treatment of rare paediatric diseases, including cystic fibrosis, primary ciliary dyskinesias and neuroblastoma. He is the founder of Nanogenic Solutions Ltd, a UCL spin-out company commercialising siRNA and gene delivery formulations.
New genetic therapies for rare diseases: from gene replacement to gene repair
Professor Stephen Hart talks about the importance of recent advances in gene therapy, including the editing of the genome to repair mutations from larger regions of DNA down to the level of single bases, as well as correcting single bases in RNA molecules