Featured Research

from universities, journals, and other organizations

Toward making the blind see: Gene therapy restores vision in mice

Date:
March 31, 2010
Source:
Federation of American Societies for Experimental Biology
Summary:
Scientists made a huge step toward making the blind see, and they did it by using a form of gene therapy that does not involve the use of modified viruses. Scientists used a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa.

Top: Normal vision. Bottom: The same scene as it might be viewed by a person with retinitis pigmentosa.
Credit: National Eye Institute, National Institutes of Health

Scientists from Buffalo, Cleveland, and Oklahoma City made a huge step toward making the blind see, and they did it by using a form of gene therapy that does not involve the use of modified viruses. In a research report published in the April 2010 print issue of The FASEB Journal, scientists describe how they used a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa, an inherited disease characterized by progressive vision loss and eventual blindness.

Related Articles


"We hope the results of our study will be instrumental in generating a cure for the debilitating blindness associated with retinitis pigmentosa and other inherited and acquired retinal diseases," said Muna I. Naash, Ph.D., a researcher involved in the work from the Department of Cell Biology at the University of Oklahoma Health Sciences Center in Oklahoma City. "Compacted DNA nanoparticles are an exciting treatment strategy for these diseases and we look forward to exciting new developments."

To make this discovery, Naash and colleagues used groups of mice with the retinal degeneration slow (Rds) gene, which causes retinitis pigmentosa. The mice received one of three types of "treatments:" nanoparticles containing the normal copy of the Rds gene, the normal gene alone, or saline solution. After these treatments were delivered to the mice, the structure and function of the retina were analyzed by comparing them to untreated mice with retinitis pigmentosa and healthy mice with the normal Rds gene. Researchers also measured the level and pattern of Rds gene expression, as well as functional, structural and biochemical improvements in disease symptoms. They discovered that mice receiving the nanoparticle gene therapy show significant signs of healing. These mice had structural improvement in their retinas, as well as functional vision improvements, which lasted throughout the duration of the study. The mice that received the gene alone or saline continued to lose their vision. The nanoparticles were safe and well-tolerated with no adverse effects.

"Making the blind see was once called a miracle," said Gerald Weissmann, M.D., Editor-in-Chief of The FASEB Journal. "As we have expanded our understanding of evolution, genetics, and nanotechnology, chances are that "miraculous" cures will become as commonplace as those claimed by faith-healers past and present."

According to the National Institutes of Health Office of Rare Diseases Research, retinitis pigmentosa is a group of inherited eye diseases that affect the retina. Retinitis pigmentosa causes cells in the retina to die prematurely, eventually leading to vision loss. There is no cure.


Story Source:

The above story is based on materials provided by Federation of American Societies for Experimental Biology. Note: Materials may be edited for content and length.


Journal Reference:

  1. X. Cai, S. M. Conley, Z. Nash, S. J. Fliesler, M. J. Cooper, M. I. Naash. Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa. The FASEB Journal, 2010; 24: 1178-1191 DOI: 10.1096/fj.09-139147

Cite This Page:

Federation of American Societies for Experimental Biology. "Toward making the blind see: Gene therapy restores vision in mice." ScienceDaily. ScienceDaily, 31 March 2010. <www.sciencedaily.com/releases/2010/03/100331141623.htm>.
Federation of American Societies for Experimental Biology. (2010, March 31). Toward making the blind see: Gene therapy restores vision in mice. ScienceDaily. Retrieved December 21, 2014 from www.sciencedaily.com/releases/2010/03/100331141623.htm
Federation of American Societies for Experimental Biology. "Toward making the blind see: Gene therapy restores vision in mice." ScienceDaily. www.sciencedaily.com/releases/2010/03/100331141623.htm (accessed December 21, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, December 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Best Tips to Curb Holiday Carbs

The Best Tips to Curb Holiday Carbs

Buzz60 (Dec. 19, 2014) It's hard to resist those delicious but fattening carbs we all crave during the winter months, but there are some ways to stay satisfied without consuming the extra calories. Vanessa Freeman (@VanessaFreeTV) has the details. Video provided by Buzz60
Powered by NewsLook.com
Sierra Leone Bikers Spread the Message to Fight Ebola

Sierra Leone Bikers Spread the Message to Fight Ebola

AFP (Dec. 19, 2014) More than 100 motorcyclists hit the road to spread awareness messages about Ebola. Nearly 7,000 people have now died from the virus, almost all of them in west Africa, according to the World Health Organization. Video provided by AFP
Powered by NewsLook.com
Researchers Test Colombian Village With High Alzheimer's Rates

Researchers Test Colombian Village With High Alzheimer's Rates

AFP (Dec. 19, 2014) In Yarumal, a village in N. Colombia, Alzheimer's has ravaged a disproportionately large number of families. A genetic "curse" that may pave the way for research on how to treat the disease that claims a new victim every four seconds. Duration: 02:42 Video provided by AFP
Powered by NewsLook.com
The Best Protein-Filled Foods to Energize You for the New Year

The Best Protein-Filled Foods to Energize You for the New Year

Buzz60 (Dec. 19, 2014) The new year is coming and nothing will energize you more for 2015 than protein-filled foods. Fitness and nutrition expert John Basedow (@JohnBasedow) gives his favorite high protein foods that will help you build muscle, lose fat and have endless energy. Video provided by Buzz60
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