Navigation Links
First clinical trial of gene therapy for childhood blindness

The first clinical trial to test a revolutionary treatment for blindness in children has been announced by researchers at UCL (University College London). The trial, funded by the Department of Health, is the first of its kind and could have a significant impact on future treatments for eye disease.

The trial involves adults and children who have a condition called Leber’s congenital amaurosis (LCA), which is a type of inherited retinal degeneration. This disease causes progressive deterioration in vision, due to an abnormality in a particular gene called RPE65. This defect prevents normal function of the retina, the light-sensitive layer of cells at the back of the eye. This results in severely impaired vision from a very young age and there are currently no effective treatments available.

The new technique that will be used in the trial involves inserting healthy copies of the gene into the cells of the retina to help them to function normally. Restoring the activity in these cells should restore vision. The operation delivers the normal genes to the retina, using a harmless virus or "vector" to carry the gene into the cells. The vector has been manufactured for this trial by Targeted Genetics, Seattle, USA.

Previous work using animal models has demonstrated that this gene therapy can improve and preserve vision. During trials, the vision of dogs with the defect was restored to the extent that they were able to walk through a maze without difficulty; something they could not do before the treatment. As this trial is the first to treat an eye disease using administration of gene therapy vectors to human retinas, the team have carried out extensive pre-clinical testing. The purpose of this trial is to find out how safe and effective the new intervention is in humans.

The team conducting the trial, from UCL Institute of Ophthalmology and Moorfields Eye Hospital, is led by Professor Robin Ali and includes leading eye surgeon Mr James Bainbridge and leading retinal specialist Professor Tony Moore.

Professor Ali said: "We have been developing gene therapy for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory. Testing it for the first time in patients is very important and exciting, and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions."

The trial’s first operations have already taken place in young adult patients who developed the condition as children. Mr James Bainbridge, who leads the surgical team, said: "It is very encouraging that we can deliver genes to an extremely fragile site in the eye without complications."

Professor Moore said: "Some indications of the results of the trial may be available within several months. However, the subjects will need to be followed-up to assess the long term effect of the treatment. It will be many months before we have the full picture. We anticipate the best outcome in younger patients, as we will be treating the disease in the early stages of its development."

Professor Ali added: "There are many forms of retinal degeneration, meaning the use of gene therapy treatments must be individually developed then tested in a separate clinical trial specifically for that disease. However, the results from this first human trial are likely to provide an important basis for many more gene therapy protocols in the future, as well as potentially leading to an effective treatment for a rare but debilitating disease."
'"/>

Source:University College London


Related biology news :

1. Timing is everything: First step in protein building revealed
2. Emory Eye Center Implants Its First Retinal Chips In Patients With Retinitis Pigmentosa
3. First atlas of key brain genes could speed research on cancer, neurological diseases
4. First-ever Compounds To Target Only Metastatic Cells Are Highly Effective Against Breast, Prostate, And Colon Cancers
5. NYCs First Rapid HIV Drug-resistant AIDS Case Prompts Call to Step Up HIV Prevention
6. First real-time view of developing neurons reveals surprises, say Stanford researchers
7. Breast-Cancer Risk Linked to Exposure to Traffic Emissions at Menarche, First Birth
8. Mayo Clinic Researchers Create Obedient Virus; First Step To Use Measles Virus Against Cancer
9. First frozen egg baby born in Canada
10. Human Cells Filmed Instantly Messaging for First Time
11. First North American Encapsulated Islet Transplant without Long-term Immune Suppression into a Patient with Type 1 Diabetes

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:3/9/2016)... , March 9, 2016 This BCC Research ... states of the RNA Sequencing (RNA Seq) market for ... as instruments, tools and reagents, data analysis, and services. ... segments of the RNA-Sequencing market such as RNA-Sequencing tools ... the main factors affecting each segment and forecast their ...
(Date:3/9/2016)... GARDENS, Fla. , March 9, 2016 /PRNewswire/ ... management authentication and enrollment solutions, today announced the ... DigitalPersona ® Altus multi-factor authentication platform. ... and InfoSec managers to step-up security where it,s ... Washington, DC . ...
(Date:3/3/2016)...  FlexTech, a SEMI Strategic Association Partner, awarded five ... Development, Leadership in Education, and, in a category new ... year of the FLEXI Awards and the winners ... past years . Judging was done on a ... criteria, by a panel of non-affiliated, independent, industry experts. ...
Breaking Biology News(10 mins):
(Date:5/25/2016)... ... May 25, 2016 , ... Thailand’s Board of Investment’s ... in San Francisco. Located at booth number 7301, representatives from the Thai Government, ... discuss the Thai biotechnology and life sciences sector. , Deputy Secretary General ...
(Date:5/25/2016)... ... ... Scientists at the University of Athens say they have evidence that the variety ... that could lead to one good one. Surviving Mesothelioma has just posted an article ... team evaluated 98 mesothelioma patients who got a second kind of drug ...
(Date:5/24/2016)... , ... May 24, 2016 , ... ... diabetes, and traumatic injuries, will be accelerated by research at Worcester Polytechnic Institute ... engines of wound healing and tissue regeneration. , The novel method, developed by ...
(Date:5/23/2016)... ... ... The need for blood donations in South Texas and across the nation is growing. , ... Center, blood donations are on the decline. In fact, donations across the country are at ... South Texas in the last four years alone. , There is no substitute for blood. ...
Breaking Biology Technology: