Toward Making the Blind See: Gene Therapy Restores Vision in Mice

G'day ATS,

I read this and I really felt hopeful that one day this could lead to a cure for blindness. This makes a welcome change from all the doom and gloom that usually greets us on the boards so I thought I'd share it with you all.

ScienceDaily

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.

Very encouraging news.

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.

What strikes me is that the treatment prompted healing rather than just stopping the disease.

This may one day lead to human cures for Retinitis pigmentosa or other forms of blindness.

All the best ATS! Kiwi


[edit on 31-3-2010 by kiwifoot]

Couldnt believe this when I read it; my father and I have retinitis pigmentosa; all I can say, thank our great Infinite Creator for your excellent vision, granted you might have something worse. This tripped me out... hope after all. I can have hope along with my faith. Thanks for posting; got my blessings, a star (wish i could give more, lol) and a flag! Thank you VERY much...

reply to post by RadiatorOfTheLight


How wonderful that this may help you and your father. Fingers crossed for this treatment to go all the way!

All the best my friend, Kiwi!