Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Making the blind see: Gene therapy restores vision in mice

01.04.2010
New research in the FASEB Journal shows that nucleic acid nanoparticle platform delivery technology achieves successful gene transfer and reverses affects of retinitis pigmentosa in mice

Take a look at this: 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 (http://www.fasebj.org), 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.

"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.

Receive monthly highlights from The FASEB Journal by e-mail. Sign up at http://www.faseb.org/fjupdate.aspx. The FASEB Journal (http://www.fasebj.org) is published by the Federation of the American Societies for Experimental Biology (FASEB). The journal has been recognized by the Special Libraries Association as one of the top 100 most influential biomedical journals of the past century and is the most cited biology journal worldwide according to the Institute for Scientific Information.

FASEB comprises 23 societies with more than 90,000 members, making it the largest coalition of biomedical research associations in the United States. FASEB enhances the ability of scientists and engineers to improve—through their research—the health, well-being and productivity of all people. FASEB's mission is to advance health and welfare by promoting progress and education in biological and biomedical sciences through service to our member societies and collaborative advocacy.

Details: Xue Cai, Shannon M. Conley, Zack Nash, Steven J. Fliesler, Mark J. Cooper, and Muna I. Naash. Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa. FASEB J. 2010 24: 1178-1191. DOI: 10.1096/fj.09-139147; http://www.fasebj.org/cgi/content/abstract/24/4/1178

Cody Mooneyhan | EurekAlert!
Further information:
http://www.faseb.org

More articles from Medical Engineering:

nachricht Novel PET tracer identifies most bacterial infections
06.10.2017 | Society of Nuclear Medicine and Molecular Imaging

nachricht Teleoperating robots with virtual reality
05.10.2017 | Massachusetts Institute of Technology, CSAIL

All articles from Medical Engineering >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Breaking: the first light from two neutron stars merging

Seven new papers describe the first-ever detection of light from a gravitational wave source. The event, caused by two neutron stars colliding and merging together, was dubbed GW170817 because it sent ripples through space-time that reached Earth on 2017 August 17. Around the world, hundreds of excited astronomers mobilized quickly and were able to observe the event using numerous telescopes, providing a wealth of new data.

Previous detections of gravitational waves have all involved the merger of two black holes, a feat that won the 2017 Nobel Prize in Physics earlier this month....

Im Focus: Smart sensors for efficient processes

Material defects in end products can quickly result in failures in many areas of industry, and have a massive impact on the safe use of their products. This is why, in the field of quality assurance, intelligent, nondestructive sensor systems play a key role. They allow testing components and parts in a rapid and cost-efficient manner without destroying the actual product or changing its surface. Experts from the Fraunhofer IZFP in Saarbrücken will be presenting two exhibits at the Blechexpo in Stuttgart from 7–10 November 2017 that allow fast, reliable, and automated characterization of materials and detection of defects (Hall 5, Booth 5306).

When quality testing uses time-consuming destructive test methods, it can result in enormous costs due to damaging or destroying the products. And given that...

Im Focus: Cold molecules on collision course

Using a new cooling technique MPQ scientists succeed at observing collisions in a dense beam of cold and slow dipolar molecules.

How do chemical reactions proceed at extremely low temperatures? The answer requires the investigation of molecular samples that are cold, dense, and slow at...

Im Focus: Shrinking the proton again!

Scientists from the Max Planck Institute of Quantum Optics, using high precision laser spectroscopy of atomic hydrogen, confirm the surprisingly small value of the proton radius determined from muonic hydrogen.

It was one of the breakthroughs of the year 2010: Laser spectroscopy of muonic hydrogen resulted in a value for the proton charge radius that was significantly...

Im Focus: New nanomaterial can extract hydrogen fuel from seawater

Hybrid material converts more sunlight and can weather seawater's harsh conditions

It's possible to produce hydrogen to power fuel cells by extracting the gas from seawater, but the electricity required to do it makes the process costly. UCF...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

ASEAN Member States discuss the future role of renewable energy

17.10.2017 | Event News

World Health Summit 2017: International experts set the course for the future of Global Health

10.10.2017 | Event News

Climate Engineering Conference 2017 Opens in Berlin

10.10.2017 | Event News

 
Latest News

Study suggests oysters offer hot spot for reducing nutrient pollution

17.10.2017 | Life Sciences

Breaking: the first light from two neutron stars merging

17.10.2017 | Physics and Astronomy

World first for reading digitally encoded synthetic molecules

17.10.2017 | Life Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>