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From Mary Kugler, R.N., for About.com

Gene therapy prevents blindness in mice with retinoschisis

Friday September 9, 2005
Scientists from the University of Florida Genetics Institute have successfully used gene therapy in mice to treat retinoschisis, an inherited disease which causes loss of sight in boys. The RS1 gene is associated with retinoschisis in humans. The researchers injected a healthy version of the human RS1 gene into the right eyes of 15-day-old male mice with retinoschisis. Six months later, the treated eyes appeared healthy and spared from the disease. The treatment has promising implications for other genetic eye diseases, including retinitis pigmentosa. The research was published in the August 2005 issue of Molecular Therapy.

Comments

June 26, 2007 at 10:01 am
(1) Melinda Bernal says:

What is the status on this research? Have additional studies been done or are they currenty being done? How about human studies? I’d appreciate any updated information. Thanks.

July 30, 2007 at 7:44 pm
(2) iswar says:

I have retinoschisis. its good to hear that the exercise went fine. Any information from anybody is welcome if there is any solution to this..

December 5, 2007 at 7:31 pm
(3) Dario D'Addio says:

Hello,
i’m affected by a retinoschisis juvenile
i’m 23 years old I come from italy, Naples.
I would like to hear from other news about this disease.

January 20, 2008 at 6:33 am
(4) John says:

I too, have Juvenile X-linked Retinoschisis as all of my brothers do. One brother has good vision at (20/40)in both eyes, and one is legaly blind at (20/500) and (20/200) I am 42 years old and am starting to have more issues with my Macula.(20/80) and (20/200) My family, and I want to know of any news from these trials, or even to join a trial. How can we help?

May 6, 2008 at 12:39 am
(5) J.Wong says:

Promising, I hope there will be more news about this!

June 16, 2008 at 9:02 am
(6) Klara says:

My sister (29 years old) has juvenile retinoschisis in both eyes. She has undergone a surgery to put her retina back and is is going to have the second one to eliminate the cyst. I’ve seen that there were some results in mice, is it possible that in the near future a trial on people would be allowed? I would appreciate any answer!!! Thank you

December 3, 2008 at 11:11 pm
(7) Trevor says:

I am very interested in this research. How can one get on a clinical study for this?

March 11, 2009 at 8:20 pm
(8) Kate says:

My son, age 31, also has hereditary Juvenile Retinoschisis. I am very interested in the latest on this gene therapy.

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