Featured Research

from universities, journals, and other organizations

New Gene Therapy Tools For Inherited Blindness Identifed

Date:
July 27, 2007
Source:
Washington University School of Medicine
Summary:
An improved approach to gene therapy may one day treat some of the nearly 200 inherited forms of blindness, scientists recently suggested. Researchers take initial steps toward filling a gap in the toolkit for treating blindness by identifying DNA elements that control when and where genes linked to blindness are turned on.

An improved approach to gene therapy may one day treat some of the nearly 200 inherited forms of blindness, scientists at Washington University School of Medicine in St. Louis recently suggested.

In a paper published online by Public Library of Science One, researchers take initial steps toward filling a gap in the toolkit for treating blindness by identifying DNA elements that control when and where genes linked to blindness are turned on.

"In its most basic form, gene therapy requires three components," explains Joseph Corbo, M.D., Ph.D., assistant professor of pathology and immunology and senior author of the paper. "You need to identify the gene that's mutated in patients and obtain a healthy copy of that gene; you need an agent to deliver the healthy gene to a patient's cells, which is usually a weakened virus; and then you also need additional DNA elements that turn on the healthy gene in the right cells and at the right levels."

Corbo and colleagues used a computational analysis of DNA to look for elements that can turn on genes in the eye's light-sensing photoreceptor cells. Their search identified hundreds of potential cis-regulatory elements, which are segments of DNA involved in turning genes on and off. They confirmed 19 of these elements, more than doubling the number known to scientists. The newly discovered elements can be used as switches to activate blindness therapies.

"This result is significant because the number of cis-regulatory elements previously available for gene therapy was quite limited," Corbo explains.

By some estimates, photoreceptors are the most energy-intensive cells in the human body, consuming more power than any other cell type. Unfortunately, high energy also brings a high degree of volatility.

"If anything goes wrong in these cells, they're going down the tubes," Corbo says. "That's why there are so many inherited forms of blindness linked to degeneration of photoreceptors." Corbo and colleagues began their studies with three genes previously shown to be important to regulation of photoreceptor proteins. The three genes, known as transcription factors, act by binding to DNA in a way that turns other genes on or off.

"Using a computerized search of DNA, we were able to identify several hundred potential binding sites for these transcription factors," Corbo explains.

Through a variety of experiments, Corbo's group confirmed that 19 of the new sites are involved in regulation of photoreceptor genes linked to inherited forms of blindness. By studying new and previously established sites, they derived some basic rules or "grammar" that seem to govern how the sites work.

"We've by no means finished defining this grammar--it's going to be a major task," he explains. "But what we know now has allowed us to create entirely synthetic cis-regulatory elements which function in photoreceptors. A more complete grammar may even one day allow us to design customized versions of cis-regulatory elements to incorporate into gene therapy vectors for individual patients."

In addition to further studies of the grammar of regulatory sites active in photoreceptor cells, Corbo and others are currently using the results of the study to design gene therapy vectors for a form of Leber's congenital amaurosis, an inherited disorder that leaves patients blind from birth.

Reference: Hsiau TH-C, Diaconu C, Myers CA, Lee J, Cepko CL and Corbo JC. The cis-regulatory logic of the mammalian photoreceptor transcriptional network. Public Library of Science One, July 25, 2007.

Funding from the National Institutes of Health supported this research.


Story Source:

The above story is based on materials provided by Washington University School of Medicine. Note: Materials may be edited for content and length.


Cite This Page:

Washington University School of Medicine. "New Gene Therapy Tools For Inherited Blindness Identifed." ScienceDaily. ScienceDaily, 27 July 2007. <www.sciencedaily.com/releases/2007/07/070726085919.htm>.
Washington University School of Medicine. (2007, July 27). New Gene Therapy Tools For Inherited Blindness Identifed. ScienceDaily. Retrieved July 30, 2014 from www.sciencedaily.com/releases/2007/07/070726085919.htm
Washington University School of Medicine. "New Gene Therapy Tools For Inherited Blindness Identifed." ScienceDaily. www.sciencedaily.com/releases/2007/07/070726085919.htm (accessed July 30, 2014).

Share This




More Health & Medicine News

Wednesday, July 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Health Insurers' Profits Slide

Health Insurers' Profits Slide

Reuters - Business Video Online (July 30, 2014) Obamacare-related costs were said to be behind the profit plunge at Wellpoint and Humana, but Wellpoint sees the new exchanges boosting its earnings for the full year. Fred Katayama reports. Video provided by Reuters
Powered by NewsLook.com
Concern Grows Over Worsening Ebola Crisis

Concern Grows Over Worsening Ebola Crisis

AFP (July 30, 2014) Pan-African airline ASKY has suspended all flights to and from the capitals of Liberia and Sierra Leone amid the worsening Ebola health crisis, which has so far caused 672 deaths in Guinea, Liberia and Sierra Leone. Duration: 00:43 Video provided by AFP
Powered by NewsLook.com
At Least 20 Chikungunya Cases in New Jersey

At Least 20 Chikungunya Cases in New Jersey

AP (July 30, 2014) At least 20 New Jersey residents have tested positive for chikungunya, a mosquito-borne virus that has spread through the Caribbean. (July 30) Video provided by AP
Powered by NewsLook.com
Generics Eat Into Pfizer's Sales

Generics Eat Into Pfizer's Sales

Reuters - Business Video Online (July 29, 2014) Pfizer, the world's largest drug maker, cut full-year revenue forecasts because generics could cut into sales of its anti-arthritis drug, Celebrex. Fred Katayama 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:
from the past week

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