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UCL EyeTherapy Blog


A blog by the Gene and Cell Therapy Group at the UCL Institute of Ophthalmology Department of Genetics


Archive for the 'Beyond the Headlines' Category

2 Lazy 2 Run? We’re Biking it for Blood Cancer!

Andi MSkilton29 August 2014


2 Lazy 2 Run Cycling Club

2 Lazy 2 Run CC (left to right): Paul Waldron, Peter Gardner, Sander Smith, Katlyn Green, Manjit Mehat, Matteo Rizzi and Sophia-Martha kleine Holthaus

On Sunday 31 August a group of not so elite athletes from the Gene and Cell Therapy group will be taking part in the London Bikeathon 2014 to raise funds for Leukaemia & Lymphoma Research.

The 2 Lazy 2 Run CC will be cycling 52 miles – that’s more than a marathon, no mean feet given that many of them haven’t cycled for some time. No wonder they’re not running!

The money raised will go towards supporting Leukaemia & Lymphoma Research who are the UK’s leading charity dedicated to improving the lives of patients with all types of blood cancer including leukaemia, lymphoma and myeloma.

Leukaemia & Lymphoma Research first started research into blood cancers in 1960 and your support means they can continue their ground-breaking research, which benefits patients today – and in the future.

If you would like to support 2 Lazy 2 Run CC then please visit their fundraising page below and donate as little or as much as you wish:

You can also find out more about Leukaemia & Lymphoma Research on their website:

The Art of Eyes

Andi MSkilton7 August 2014

Embryonic stem cell-derived photoreceptors

The eye is an object of great beauty as shown by the Ophthalmologist in their July/August 2014 issue. This month’s issue features a photo essay called The Art of the Eyes and includes examples of the work from a number research labs capturing the complex and beautiful detail of the eye and its cells. The essay includes images of embryonic stem-cell-derived photoreceptors produced by our own Dr Colin Chu and Dr Anai Gonzales-Cordero, who’s work graces the cover (pictured).

In memoriam

Andi MSkilton5 August 2014


Dr Yoshiki Sasai (1962 – 2014)

It is with great sadness today that we remember and pay tribute to our collaborator Dr Yoshiki Sasai.
Yoshiki was a world leading stem cell researcher and Deputy Director of the Riken Center for Developmental Biology in Kobe, Japan.
Through his hard work and dedication over many years, Yoshiki made an enormous contribuution to the field of regenerative medicine, which remain as a lasting testament to an accomplished scientist. 
We will remember Yoshiki fondly and with respect and our thoughts are with his family, friends and colleagues during this difficult time.

Fun and Learning Had by All at the BSGCT Public Engagement Day!

Andi MSkilton14 March 2014

Screen Shot 2014-03-13 at 16.13.47

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Last week, Fri 7 March, was the public engagement day of the British Society for Gene and Cell Therapy (BSGCT) at the Oxford University Museum of Natural History.

Dr Tassos Georgiadis, UCL Institute of Ophthalmology has been on the board of the BSGCT since 2012 and chairs the public engagement sub-committee. The day provided the public with an opportunity to hear from a prestigious panel of scientists working at the forefront of gene and stem cell research as well as an opportunity for students to learn more about what it means to be a scientist.

“The day was a great success and everyone involved has said they really enjoyed it,” said Dr Georgiadis. “We had an eminent speaker panel who were able to talk from their own experiences of working on gene and cell therapies and to dispel many of the myths and misreporting we hear everyday. The audience seemed to really appreciate a chance to learn and hear first-hand why these are such challenging and time intensive treatments to develop.”

As well as getting to hear about cutting edge research there was the opportunity for attendees to meet, discuss and debate with scientists, patients, journalists and clinicians around the impact that research into genetic and stem cell therapies has for society.

It was extremely engaging to watch the public as well as GCSE and A-Level students grill the speakers with some highly challenging questions around the technology and ethics behind gene and stem cell therapies. And Adam Pearson, a Patron of Genetic Disorders UK, gave a really enjoyable and honest account of his own experience of living with a genetic condition and the need to ensure that researchers and clinicians keep the person and not the disease at the forefront of their mind.

Our congratulations go out to everyone involved, for a truly fantastic and inspiring event!

The Department of Genetics, UCL Institute of Ophthalmology will be holding their own public engagement day on 5 July 2014 for people with age-related macular degeneration (funded by the NIHR BRC Moorfields Eye Hospital NHS Trust, the Macular Society and by a grant from the Wellcome Trust).

You will have the opportunity to share your experiences of AMD with charities, researchers and healthcare professionals. You will hear first-hand the progress being made in world-leading research into gene and stem cell therapies for AMD and other forms of macular degeneration and have a chance to discuss the focus for future research to enable us to further support the needs and aspirations of people living with AMD.

Check back at the end of March 2014 for information on how to register to attend.  

A New Viral Vector with the Potential to Improve Eye Gene Therapy

PrateekBuch28 June 2013


A new type of viral vector has been developed using an innovative research technique, by researchers at the University of California, Berkeley. The new virus shows great promise as a tool for delivering genes to the eye, because it has the potential to deliver genes to the retina when injected into the gel of the eye (called the vitreous), whereas current eye gene therapy vectors have to be injected under the retina using a more invasive approach in order to reach the target cells. The new vector was reported in the media as a ‘wonder jab’ that could ‘cure blindness in 15 minutes,’ but a closer examination of its effectiveness shows there is a long way to go before such a vector could be effective in the clinic.

While the new vector was shown to deliver genes very efficiently to the light sensitive photoreceptor cells and supporting RPE cells in the mouse eye when injected into the vitreous gel, experiments in non-human primates suggest that there might be further barriers to overcome before the new vector can be considered for use in clinical trials.

The new virus is based on the same adeno-associated virus (AAV) that we are using in our clinical trial of gene therapy for Leber congenital amaurosis (LCA) – in this study David Schaffer and colleagues took the most commonly-used type of AAV and altered it in a number of ways. They were looking to develop a virus that would deliver genes to the light sensitive photoreceptor cells at the back of the eye after an injection into the vitreous jelly at the front of the eye.

The approach they took was to introduce variation into the protein coat of AAV – which is what determines the type of cell the virus delivers genes to, and how efficiently it does so. They did this by introducing a random protein sequence into the virus coat, or by shuffling protein sequences with other types of AAV. The investigators injected these randomly-altered AAV particles, and a week later harvested cells that the vector had successfully delivered DNA to (which Dr. Schaffer’s team could identify as the gene being delivered was for green fluorescent protein, GFP). This allowed the team to see which of the new variants could deliver genes to cells in the eye – by repeating the process, the team identified the most efficient AAV variant. This process of directed evolution – selecting randomly-altered viruses for their ability to deliver genes to cells in the retina – produced dozens of new AAVs capable of targeting photoreceptor cells. Dr. Schaffer’s team used one variant for further testing in models of sight loss.

The new AAV – called 7m8 –efficiently delivered DNA to all cell types in the mouse retina when injected into the vitreous gel. When engineered to deliver therapeutic genes, it also restored vision in mouse models of retinoschisis and of LCA – inherited conditions caused by mutations in the Rsh1 and RPE65 genes respectively.

Dr. Schaffer’s team then used the new 7m8 vector in monkey eyes, to test how well it could deliver genes to a non-human primate retina following an injection into the front of the eye. Here, they saw that whilst the new virus could deliver the GFP gene to some photoreceptor cells, the vector was not nearly as efficient in monkey as in mouse. There was patchy gene delivery, suggesting that photoreceptor cells in the monkey eye, which has many features in common with the human eye, are harder to reach following injection into the vitreous gel – even when using the new type of vector.

The aim of this research was to develop a new type of AAV – using directed evolution to generate lots of variations and selecting the best one – that could deliver genes to the retina using a less invasive approach that would be faster and less risky than the sub-retinal surgery that is currently used. Dr Schaffer’s vector appears to be the most efficient way of delivering genes to the mouse retina when injected into the vitreous, an approach that has been tried with less efficiency by other labs in previous studies. If this new vector can be further modified to be efficient in larger eyes that resemble the human eye, then it has promise for clinical application – but it is far from the ‘wonder jab’ that it was reported to be by some in the media. It is an improved tool to deliver genes, but in itself the new vector can’t be considered a treatment. We’ll need to watch this space, as further research is required to see if this tool can lead to more effective treatments.

Stem Cell Therapies for Sight Loss – How to Avoid Bogus Treatments

PrateekBuch4 February 2013

Stem cells – cells that have the capacity to turn into virtually any cell in the human body – are an exciting source of potential treatments for a huge range of medical conditions. Stem cells are already being used in the treatment of disorders of the blood. Academics, clinical specialists and companies throughout the world are developing treatments for other conditions, including many that cause sight loss. Our own group has begun testing the safety of transplanting retinal cells grown from stem cells in patients with Stargardt disease.

Unfortunately there are a number of ‘stem cell therapies’ that are being promoted as proven treatments, without scientific evidence or appropriate approval. Here we aim to help those considering stem cell therapy for sight loss to distinguish between the approved experimental techniques in clinical trials, and the bogus treatments being offered elsewhere.

It’s important to remember that no stem cell treatment has been approved for eye disease by the medical authorities. This is because clinical trials involving stem cells in the eye are at the very earliest stages, and it is too early to know whether their use is safe or effective. Until such trials are completed, any treatments on offer are likely to be unproven.

There are a number of clinics and companies that offer what they describe as stem cell transplants for a wide range of conditions, often at significant cost. These transplants have not been subject to the rigorous safety checks that are part of properly-conducted clinical trials. The clinics offering them are usually located in countries with less stringent standards of safety and regulations than we expect in the UK, enabling them to sell such treatments in the absence of published scientific evidence to support them.

The treatment typically involves the injection of stem cells (or rather cells that come from bone marrow, or the umbilical cords of newborn babies) without first turning them into a particular cell type. The difference between these cells and cells we are using in our clinical trial is a subject for another day, but for now it’s worth remembering that the cells in our studies are first made into retinal cells in a dish before being delivered into the eye. There is no evidence that stem cells injected into the body can reach the eye, or that once they do they can repair any damage. Furthermore, there is a very real possibility that such cells could cause harm by proliferating uncontrollably.

Turning stem cells into retinal cells in a dish

In our cell transplantation clinical trial we take stem cells and turn them into retinal cells in a dish – it is these retinal cells, not the stem cells themselves, that we inject. The injection of stem cells remains an unproven and potentially dangerous technique.

Companies offering these injections often feature patient testimonials, which are not a reliable indicator of safety or efficacy. The only reliable, peer-reviewed, published account of a patient receiving such transplants that we are aware of shows very clearly that there was no way to tell what kind of cells were injected, and that there was no effect whatsoever on vision after treatment.

In the absence of any scientific evidence supporting the direct injection of stem cells, we cannot recommend any of the treatments currently on offer. Should you be considering any such treatments, we strongly recommend that you check our Frequently Asked Questions and contact us first for advice.

Aspirin and AMD – Beyond the Headlines

PrateekBuch24 January 2013


A number of recent media stories have highlighted two studies examining the link between long-term use of aspirin and the risk of developing the “wet” form of age-related macular degeneration (AMD). Because some of the headlines are misleading, we would urge you to consider the following details about the studies and the advice that follows.

In a study published last December in the Journal of the American Medical Association, specialists at the University of Wisconsin followed the vision of nearly 5000 patients over a period of fifteen years. They found that those who were prescribed daily aspirin to protect against heart disease were more likely to develop the “wet” form of AMD (in which the growth of new blood vessels damages the retina) than those who didn’t take aspirin. However, the risk of developing wet AMD rose from around 1-in-100 to just over 1-in-60 – which is a small difference in a rare risk.

A study from the University of Sydney, published in January 2013 in the separate journal JAMA Internal Medicine, showed that 4% of people who did not take aspirin regularly developed wet AMD, whilst 9% of those who took aspirin daily for ten years developed the condition.

While these studies suggest an association between long-term aspirin use and increased risk of AMD, they do not tell us whether any relationship is causative. Previous studies looking at the link between aspirin and AMD have been inconclusive. There is not yet enough evidence to know whether the increased risk of wet AMD is caused by aspirin itself or by other health factors for which aspirin is used.

Commenting on the media stories linking aspirin and AMD, Professor James Bainbridge said, “Daily aspirin use is well-established as an effective way to protect against heart disease in people at high risk. We therefore support the advice given by the Macular Society and others, which advises those who take aspirin to lessen their risk of heart attacks to continue to do so. Given that wet AMD can be managed using drug therapy, and that aspirin use can significantly reduce the risk of heart disease, there is no need as yet to change aspirin use based on these studies.”