Today we released a joint statement with other medical research funders in support of research using gene editing techniques such as CRISPR-Cas9 to advance our understanding of disease in preclinical research and to explore their potential for future therapeutic use. Here our Director of Science Programmes Dr Rob Buckle discusses the huge potential of gene editing, and why any attempt to use it in a therapeutic context must be the subject of the kind of intense and rigorous debate that the scientific community and UK regulatory system has demonstrated in the past.
Being able to edit the human genome is not a new idea or capability. For decades researchers have been developing potential gene therapy techniques to correct missing or faulty DNA and restore healthy gene expression in cells.
More recently, techniques have been developed that can edit the genome in a much more efficient and targeted fashion, and one such example, CRISPR-Cas9, has accelerated the field to such an extent that researchers can now make precise edits to the genome in a relatively easy, speedy, precise and error-free way. Read more
Professor Robin Ali is an MRC-funded scientist working at the forefront of not one, but two, fields of regenerative medicine: gene and stem cell therapy. Katherine Nightingale caught up with Robin at UCL’s Institute of Ophthalmology to find out more about his work.
The eye is fertile ground for developing new therapies, a feature that Robin Ali is taking full advantage of. Not content with a thriving gene therapy programme, he took up the challenge of entering the world of stem cell therapy in 2004 when the MRC was funding researchers to move in from other fields. Robin’s research focuses on therapies for retinal disorders, mainly those that affect the light-sensitive ‘photoreceptor’ cells of the eye. Many of these are rare, single-gene disorders that cause vision loss over time — and which currently have no treatments.
Lucky for Robin, “the properties of the eye lend it to experimental interventions,” he says. It is fairly straightforward to operate on, and the progress of a therapy — improved retinal sensitivity, for example — can be easily monitored. The eye is also somewhat protected from the body’s immune system, so there is less of an inflammatory response to introduced genes or cells. Read more