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
'"/>

Source:University College London


Page: 1 2

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:
TAG: First clinical trial gene therapy for childhood blindness

(Date:7/9/2014)... one of the amphibians with the highest distribution in ... of water where it comes into contact with the ... carried out by the Spaniard Germn Orizaola from the ... these frogs have developed a defensive response to the ... bodies if they co-exist with the crayfish. , Numerous ...
(Date:7/9/2014)... function and increase the survival rate of naturally ... School of Pharmaceutical Sciences, Southwest University, China performed ... administered with low dose of levothyroxine for 3 ... exhibited an obvious improvement in cognitive and an ... The underlying mechanism was demonstrated that levothyroxine treatment ...
(Date:7/9/2014)... vitamin D in their blood are more likely to survive ... highest levels of vitamin D have half the risk of ... reveal. , The study is the first to correlate total ... their diagnosis which includes that produced after exposure to ... long term survival prospects. , The University of Edinburgh ...
Breaking Biology News(10 mins):Frogs have developed rapid defences against the red swamp crayfish 2Frogs have developed rapid defences against the red swamp crayfish 3
... is helping to reduce the drug cravings of addicted rats. ... some parts of the brain lose glutamate receptors from the ... neurons. The researchers have sidestepped this problem by crafting a ... glutamate receptor and, once inside a neuron, serves as a ...
... discovered in tiny snow fleas by Queen's University researchers ... transplantation. , Drs. Laurie Graham and Peter Davies, from ... produced by the fleas to protect themselves against freezing ... Celsius degrees. This would allow organs to be stored ...
... zebrafish may be able to play a critical role in ... that can lead to it - and identified one gene ... , The findings were published in a professional journal, Proceedings ... State University and two Boston hospitals, the Brigham and Women's ...
Cached Biology News:Grabbing addiction by the tail 2Grabbing addiction by the tail 3New antifreeze protein found in fleas may allow longer storage of transplant organs 2Zebrafish may hold key to improved cancer research 2Zebrafish may hold key to improved cancer research 3
(Date:1/14/2014)... 2014 EquitiesIQ, a leading informational research ... Alliqua is an emerging biomedical company acquiring, developing, manufacturing, ... market. , Free report download: http://equitiesiq.com/reports/alliqua/ , ... management team and Board, which launched the company’s new ...
(Date:1/14/2014)... Boston, MA (PRWEB) January 14, 2014 iLab ... Michelle Detwiler as the new Director of Product Strategy. In ... as well as iLab sub-teams to guide in the development ... that iLab provides the maximum possible benefit to the scientific ...
(Date:1/14/2014)... Global Record Systems, LLC, (GRS), ... solutions for patients, physicians, the biopharmaceutical industry, regulators, ... the signing of a three-year Research Collaboration Agreement ... (FDA). This initiative is designed to generate ...
(Date:1/14/2014)... January 14, 2014 Kerr Corporation, a leading ... and additional how-to information about dual arch impressions on its ... Dual Arch Impressions,” the blog entry serves up a list ... houses a step-by-step demonstration by Dr. David Little as he ...
Breaking Biology Technology:EquitiesIQ Initiates Coverage of Alliqua, Inc. 2EquitiesIQ Initiates Coverage of Alliqua, Inc. 3iLab Solutions Announces Michelle Detwiler as the New Director of Product Strategy 2Global Record Systems Announces Research Collaboration Agreement with FDA to Create a Novel “Big Data” Paradigm for Collection of Patient Safety and Outcomes Information 2
... 9 The results of a new study on immune-based ... sites around the country, will be published in the December ... MD, MS, director of the Butler Hospital Memory and Aging ... Warren Alpert Medical School of Brown University, says that this ...
... , , QUEBEC CITY, Dec. 9 /PRNewswire-FirstCall/ ... AEZ) (the "Company"), a global biopharmaceutical company focused on ... appointment of Pierre Lapalme to its Board of Directors. ... has over the course of his career held numerous ...
... , OXFORD, England, December ... Recent Recruitment of Senior Antibody Industry,Leaders and Highlights OBT,s Growth ... Oxford BioTherapeutics (OBT) has today announced the appointment of ... experience to OBT at an exciting phase in the Company,s,development, ...
Cached Biology Technology:Butler Hospital Neurologist Lead Author of Ground Breaking Bio-Engineered Treatment Study for Alzheimer's Disease 2AEterna Zentaris Announces Appointment of New Board Member 2Oxford BioTherapeutics Appoints Michael Moore as Chairman 2Oxford BioTherapeutics Appoints Michael Moore as Chairman 3Oxford BioTherapeutics Appoints Michael Moore as Chairman 4
X-Gal, 1 g. 5-Bromo-4-chloro-3-indolyl-b-d-galactoside.Histochemical substrate for b-galactosidase, used in blue/white screening.Store at -20 C. Protect from moisture. Category: Nucleotides & Enzymes...
Recombinant Rat Fas Ligand/TNFSF6...
Request Info...
FGF Receptor 1 Antibody...
Biology Products: