What is gene therapy and how can it help genetic eye disease?
Join Crick Group Leader Mariya Moosajee as she explains gene therapy through the lens of her work as clinical scientist and an ophthalmologist (eye surgeon).
Gene therapy aims to treat or cure diseases by targeting the underlying genetic cause. It works by adding, removing or fixing genetic material inside the body’s cells.
Genetic eye diseases affect 1 in 1000 people, but there is currently only one gene therapy available on the NHS. This therapy can only help about 80 patients in the UK and causes significant side effects.
Mariya’s lab is developing new, potentially safer, genetic therapies that could help patients across the world with rare diseases.
Mariya is a clinician scientist, an ophthalmologist (eye surgeon) and a molecular biologist. She sees patients with rare blinding eye conditions that have a genetic basis. For example, children born with no eyes or those who go blind in the first few years of life. Mariya undertook her medical degree (MBBS) and PhD at Imperial College London, where she investigated the genetic causes of eye defects at birth.
In 2020, she joined the Crick to lead the occular genomics and therapeutics laboratory.
She is a strong advocate for women in science and has been named in the top 100 most influential people in Ophthalmology worldwide in The Ophthalmologist Power List every year since 2019.
Crick Crash Courses started life as talks for our staff, to help everyone working here, scientists and non-scientists, better understand the Crick's discovery research into how life works.
We're now opening our doors to our crash course talks, giving everyone, everywhere, the opportunity to hear directly from our scientists as they investigate some of the most complex challenges in human health.
