Gene therapy - What is it and how might it help people with cavernoma?

Gene therapy - What is it and how might it help people with #cavernoma?

Increasingly clinicians are looking to gene therapies as a #treatment for #rare conditions. For the 20% of people with the familial form of #cavernoma, gene therapy for #cavernoma may offer an important future treatment prospect. Given this, in the coming years CAUK hopes to kick start gene therapy research in the UK building on work started in the US.

Whilst we're several years away from making gene therapy a reality, we're keen to start raising awareness of what gene therapy is and the pathway to developing a novel gene therapy in the UK. This webinar will be delivered by Dr Ellie Chillcot who is a Research Fellow at UCL with experience in developing gene therapies for rare neurological conditions. Dr Chillcot will be available at the end to answer questions.

A cavernoma is made up of abnormal blood vessels and can be found in the brain and/or spinal cord and looks like a raspberry.

1 person in 600, in the UK, has a brain cavernoma without symptoms (asymptomatic). This equates to roughly 90,000 people – enough to fill Wembley Stadium. A spinal cavernoma is rarer than a brain cavernoma.

For more information about Cavernoma Alliance UK, please visit https://cavernoma.org.uk/

This is a recording of one of our webinars given by our members, guests and medical experts throughout 2023.

Видео Gene therapy - What is it and how might it help people with cavernoma? канала Cavernoma Alliance UK