Navigation Links
Making the blind see: Gene therapy restores vision in mice
Date:3/31/2010

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.


'/>"/>

Contact: Cody Mooneyhan
cmooneyhan@faseb.org
301-634-7104
Federation of American Societies for Experimental Biology
Source:Eurekalert

Related biology news :

1. Coal and black liquor can produce energy from papermaking
2. Another type of nanotube, a how-to guide to making bamboo-structured carbon nanotubes
3. GBIF making the search for biodiversity research resources easier
4. Making sense of antisense microRNAs
5. Is that sea otter stealing your lunch -- or making it?
6. LSU scientist finds evidence of rain-making bacteria
7. Making sure the wonder materials dont become the wonder pollutant
8. Findings a step toward making new optical materials
9. Research suggests parts of UK could be too hot for wine-making by 2080
10. Research suggests parts of UK could be too hot for wine making by 2080
11. Small protein may have big role in making more bone and less fat
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/17/2016)... 17, 2016 Global Market Watch: Primarily ... Banks, Population-Based Banks and Academics) market is to witness a ... Biobanks shows the highest Compounded Annual Growth Rate (CAGR) of ... during the analysis period 2014-2020. North America ... followed by Europe at 9.56% respectively. ...
(Date:11/14/2016)... Nov. 14, 2016  xG Technology, Inc. ("xG" or ... critical wireless communications for use in challenging operating environments, ... 30, 2016. Management will hold a conference call to ... p.m. Eastern Time (details below). Key Recent ... $16 million binding agreement to acquire Vislink Communication Systems. ...
(Date:6/22/2016)... -- On Monday, the Department of Homeland Security (DHS) ... for the Biometric Exit Program. The Request for Information ... explains that CBP intends to add biometrics to confirm ... States , in order to deter visa overstays, ... Logo - http://photos.prnewswire.com/prnh/20160622/382209LOGO ...
Breaking Biology News(10 mins):
(Date:12/8/2016)... England , December 8, 2016 ... das Unternehmen für Molekulargenetik, erweitert seine Palette an ... myPanel™ NGS Custom FH Panels, das ein schnelles ... (FH) ermöglicht. Das Panel bietet eine Erkennung von ... Variations (CNV) mit einem einzigen kleinen Panel und ...
(Date:12/8/2016)... ... December 08, 2016 , ... ... commercial launch of flexible packaging for their exceptionally efficient human mesenchymal stem/stromal ... extends RoosterBio’s portfolio of bioprocess media products engineered to radically streamline culture ...
(Date:12/8/2016)... Dec. 8, 2016 Soligenix, Inc. (OTCQB: SNGX) ... on developing and commercializing products to treat rare diseases ... that it will be hosting an Investor Webcast Event ... the origins of innate defense regulators (IDRs) as a ... oral mucositis and the recently announced and published Phase ...
(Date:12/8/2016)... to fuel Philadelphia,s innovative digital ... Southeastern Pennsylvania (" Ben Franklin "); Independence ... Cross; and Safeguard Scientifics ("Safeguard") (NYSE: SFE ... funding initiative over a four year period to grow ... burgeoning economic vitality in digital health, Ben Franklin ...
Breaking Biology Technology: