Featured Research

from universities, journals, and other organizations

Successful Tests Of New Treatments In Mice For Eye Disease Causing Irreversible Blindness In Humans

Date:
November 1, 2005
Source:
Case Western Reserve University
Summary:
Researchers examined the effect of combining two treatments, gene therapy and oral medication, in blind mice that did not have the LRAT enzyme. They report these treatments "provide highly effective and complementary means for restoring retinal function in this animal model of human hereditary blindness."

A team led by Krzysztof Palczewski, Ph.D., chair of pharmacology at the Case Western Reserve University School of Medicine, has taken the first steps in treating an eye disease causing irreversible congenital blindness in millions of people worldwide by successfully testing two new treatments in mice.

Publishing in this month's open access journal PLoS Medicine, the researchers found that these treatments "provide highly effective and complementary means for restoring retinal function in this animal model of human hereditary blindness."

The disease studied is Leber congenital amaurosis (LCA), characterized by severe loss of vision at birth. Its causes are not fully understood. Researchers believe that the disease might be due to abnormal development of photoreceptor cells in the retina, extremely premature degeneration of these cells, or lack of essential metabolic ingredients necessary for vision in the cells. In a subset of these diseases, it is known that the retina stops functioning due to loss of the lecithin retinol acyl-transferase enzyme (LRAT). LRAT is required for regeneration of a pigment necessary for the eye to detect light.

LCA can be caused by mutations in the gene encoding RPE65, a key protein involved in the production and recycling of 11-cis-retinal in the eye. Currently, there is no treatment for LCA, although previous studies in mice have successfully tested the injection of a virus carrying the normal gene for RPE65, and, separately, oral administration of a vitamin A-like compound.

In the current paper, Palczewski (formerly of the University of Washington) examined the effect of combining the two treatments in blind mice that did not have the LRAT enzyme. They report that gene therapy carrying the LRAT gene significantly restored electroretinographic (ERG) responses and pupillary light responses. Pharmacological intervention with orally administered drugs also caused long-lasting restoration of retinal function in LRAT-deficient mice and increased ERG response.

They noted that the oral treatment was easier to administer compared with injecting the gene therapy directly into the eye, but a disadvantage of the oral treatment was a potential for long-term systemic toxicity compared with the gene therapy. However, toxicological data gathered in this and previous studies have suggested no long term ill effects in mice.

It is possible that each treatment might eventually prove to be more suitable for a specific age group of patients, and therefore, combining the therapies might offer more effective treatment for a wider age range of patients, suggest the authors.

The team hopes that if the treatments are used together, treatment with oral retinoids could begin in infancy to avoid early sight loss and the difficulties associated with surgery in very young patients. And when patients are older, long-lasting drug-free treatment could be done by surgically introducing gene therapy. This study marks the first step in finding out whether these treatments will work effectively and safely in humans.


Story Source:

The above story is based on materials provided by Case Western Reserve University. Note: Materials may be edited for content and length.


Cite This Page:

Case Western Reserve University. "Successful Tests Of New Treatments In Mice For Eye Disease Causing Irreversible Blindness In Humans." ScienceDaily. ScienceDaily, 1 November 2005. <www.sciencedaily.com/releases/2005/11/051101222855.htm>.
Case Western Reserve University. (2005, November 1). Successful Tests Of New Treatments In Mice For Eye Disease Causing Irreversible Blindness In Humans. ScienceDaily. Retrieved October 21, 2014 from www.sciencedaily.com/releases/2005/11/051101222855.htm
Case Western Reserve University. "Successful Tests Of New Treatments In Mice For Eye Disease Causing Irreversible Blindness In Humans." ScienceDaily. www.sciencedaily.com/releases/2005/11/051101222855.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
WHO: Ebola Vaccine Trials to Start a in January

WHO: Ebola Vaccine Trials to Start a in January

AP (Oct. 21, 2014) Tens of thousands of doses of experimental Ebola vaccines could be available for "real-world" testing in West Africa as soon as January as long as they are deemed safe in soon to start trials, the World Health Organization said Tuesday. (Oct. 21) Video provided by AP
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

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