Just Gene Therapy Lecture
Alton EW, A Lecture Organised by Just Gene Therapy, Brompton Hospital, London
2012, 09-11
The following lecture was recorded on 11 September, 2012 at Imperial College London.
The event was organised by Just Gene Therapy, the new not for profit organisation established to raise funds CF gene therapy by Rosie Barnes.
In the lecture Prof Eric Alton summaries the aims of the UK CFGTC and our progress to date.
Highlights
00:00 Rosie Barnes Introduction
01:00 Start of Lecture
06:30 DIY gene therapy
12:40 Previous clinical trials
16:40 Formation of the UKCFGTC
20:30 Improving the duration of transgene expression
23:00 Our clinical trial programme
27:50 The single dose (pilot) clinical trial
33:00 Can the formulation be repeatedly administered
35:45 The multi-dose clinical trial
37:28 Wave II - Can we do better
Just Gene Therapy Lecture, 11 September 2012(Prof Eric Alton summaries the aims of the UK CFGTC and our progress to date.)
What is Gene Therapy? (2008): CF gene therapy aims to insert a fully functional CFTR gene into the lung airway cells of people with the condition. Two main types of vector are used to deliver this new copy of the gene. A viral vector, engineered from viral particles or non-viral vectors, plasmid DNA molecules condensed by other chemical entities. More...