You may have seen in the news recently bulletins about Professor Robin Ali's published work on gene therapy for a degenerative retinal disease. 

 

'Gene Therapy for Leber's Congenital Amaurosis' -  two groups of investigators describe the first results of separate clinical trials investigating the short-term safety and preliminary efficacy of gene therapy.

 

See:  http://content.nejm.org/ New England Journal of Medicine (24th April 2008 edition) for full details of this work.

 

Professor Ali was part of the gene therapy team based at the Institute of Ophthalmology, UCL and attended our recent workshop 'Bringing Therapies to Batten disease'.  This workshop brought together for the first time in the UK c 40 researchers and clinicians to discuss Batten disease, with a view to identifying mechanisms for taking forward clinical and research work.  We therefore discussed this trial with him as part of our looking at therapy options for improving quality of life for Batten's children. 

 

In context, this research which is in it's early stages, may have beneficial implications in the next few years and the success of the latest trial is encouraging, but there are also other options to be explored in Professor Ali's current field of investigations with regard to positive outcomes for Batten Disease patients.  We hope that, by establishing an effective working relationship with Professor Ali and others at Moorfields, we can support this exciting area of work and keep our members informed of relevant developments as they emerge.