Featured Research

from universities, journals, and other organizations

Viral vector designed to treat a genetic form of blindness

Date:
November 2, 2011
Source:
Ohio State University Medical Center
Summary:
Researchers have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males. The vector is being tested in a clinical trial investigating the use of gene therapy to cure choroideremia, a disease that affects an estimated 100,000 people worldwide.

Researchers at Ohio State University Medical Center and Nationwide Children's Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males.

The vector is part of a clinical trial investigating the use of gene therapy to cure choroideremia, a disease that affects an estimated 100,000 people worldwide. The trial is being conducted by researchers at the University of Oxford in England.

The vector was designed by Dr. Matthew During, professor of molecular virology, immunology and medical genetics and of neuroscience and neurological surgery at Ohio State, in collaboration with Robert MacLaren, professor of ophthalmology at the University of Oxford, who also leads the trial.

Researcher Dr. K. Reed Clark, director of the Clinical Manufacturing Facility at the Center for Gene Therapy, Nationwide Children's Hospital, and his team produced the clinical-grade vector that is administered to patients in the trial.

During, who is also a visiting professor of translational neuroscience at Oxford, was in the operating room during the pioneering surgery. "I and my colleagues are excited about contributing to this significant medical breakthrough," During says. "We have worked for many years to engineer and optimize viruses to safely deliver genes to humans, and the eye is an ideal target in many ways. The clinical vector manufacturing facility at Nationwide Children's Hospital is outstanding, and Dr. Clark and his team deserve congratulations for providing a clinical vector that for the first time offers these patients the possibility of an effective therapy."

During and his colleagues designed the viral vector to infect the light-sensitive photoreceptor cells that line the back of the eye and make up the retina. Choroideremia causes a degeneration of these photosensitive retinal cells and progressive blindness. The diagnosis is usually made in childhood and leads to blindness by around age 45.

"This trial represents the first attempt to treat this disease and the first time that gene therapy has been directed towards the photoreceptor cells of the human retina," During says. "We believe it holds great promise for the treatment of other genetic causes of blindness such as retinitis pigmentosa."

The trial's 12 patients will be treated in one eye. It will take 24 months to know whether the gene-therapy treatment has stopped the degeneration. The trial builds on gene-therapy research performed in collaboration with Professor Miguel Seabra at Imperial College London, along with During and Clark at Ohio State.

"This disease has been recognized as an incurable form of blindness for over a hundred years," MacLaren says. "I cannot describe the excitement in thinking that we have designed a genetic treatment that could potentially stop it in its tracks with one single injection."


Story Source:

The above story is based on materials provided by Ohio State University Medical Center. Note: Materials may be edited for content and length.


Cite This Page:

Ohio State University Medical Center. "Viral vector designed to treat a genetic form of blindness." ScienceDaily. ScienceDaily, 2 November 2011. <www.sciencedaily.com/releases/2011/11/111102125357.htm>.
Ohio State University Medical Center. (2011, November 2). Viral vector designed to treat a genetic form of blindness. ScienceDaily. Retrieved October 21, 2014 from www.sciencedaily.com/releases/2011/11/111102125357.htm
Ohio State University Medical Center. "Viral vector designed to treat a genetic form of blindness." ScienceDaily. www.sciencedaily.com/releases/2011/11/111102125357.htm (accessed October 21, 2014).

Share This



More Health & Medicine News

Tuesday, October 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

CDC Revamps Ebola Guidelines After Criticism

CDC Revamps Ebola Guidelines After Criticism

Newsy (Oct. 21, 2014) The Centers for Disease Control and Prevention have issued new protocols for healthcare workers interacting with Ebola patients. Video provided by Newsy
Powered by NewsLook.com
First-Of-Its-Kind Treatment Gives Man Ability To Walk Again

First-Of-Its-Kind Treatment Gives Man Ability To Walk Again

Newsy (Oct. 21, 2014) A medical team has for the first time given a man the ability to walk again after transplanting cells from his brain onto his severed spinal cord. Video provided by Newsy
Powered by NewsLook.com
CDC Issues New Ebola Guidelines for Health Workers

CDC Issues New Ebola Guidelines for Health Workers

Reuters - US Online Video (Oct. 21, 2014) The U.S. Centers for Disease Control and Prevention has set up new guidelines for health workers taking care of patients infected with Ebola. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
'Cadaver Dog' Sniffs out Human Remains

'Cadaver Dog' Sniffs out Human Remains

AP (Oct. 21, 2014) Where's a body buried? Buster's nose can often tell you. He's a cadaver dog, specially trained to find human remains and increasingly being used by law enforcement and accepted in courts. These dogs are helping solve even decades-old mysteries. (Oct. 21) Video provided by AP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins