Gene Therapy for Retinitis Pigmentosa May Prevent Blindness

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There is good news for the estimated 100,000 Americans who live with retinitis pigmentosa. Scientists have discovered how to deliver gene therapy directly to the cells affected by this eye disease and thus prevent blindness.

The discovery was the result of a collaborative effort between researchers at the University of Oklahoma Health Sciences Center and colleagues in Cleveland and Buffalo. Muna Naash, PhD, at the University of Oklahoma, said that “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.”

Retinitis pigmentosa is a term that refers to a group of inherited diseases that cause a degeneration of the retina, the part of the eye that captures images from the visual field. People who develop retinitis pigmentosa experience a gradual decline in their vision as the cells that capture the images (photoreceptor cells called rods and cones) die. Forms of retinitis pigmentosa and related diseases include Leber’s congenital amaurosis, Usher syndrome, and rod-cone disease, among others.

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Symptoms of the disease depend on which cells are affected first. The Foundation Fighting Blindness notes that in most cases, the rods are impacted, which means individuals lose their peripheral and night vision. The cones are responsible for color and sharp central vision. Retinitis pigmentosa is usually first diagnosed during adolescence and young adulthood. Most people with the disease are legally blind by age 40.

Treatment for retinitis pigmentosa has focused on nutritional therapy, gene therapy, and implantable microchips. This latest study from the University of Oklahoma utilized nanoparticle technology to deliver genetic therapy inside cells of the retina.

The microscopic capsule created by scientists to transport the therapies has been very effective, arriving at the designated location in the eye within 15 minutes of delivery and quickly sending its contents to nearby cells. Robert E. Leonard, MD, an ophthalmologist at the Dean McGee Eye Institute called the work “an incredible breakthrough” and said that “outside of gene therapy, we are at a loss to be able to treat these patients.”

The study, which appears in the Journal of the Federation of American Societies for Experimental Biology (FASEB), is only the beginning for people with retinitis pigmentosa and others diseases. Already the discovery is being used to develop new treatments for macular degeneration, the leading cause of blindness in the United States. The microscopic capsule has also been tested in animals with the potential of treating diseases ranging from bladder cancer to diabetes.

SOURCES:
Foundation Fighting Blindness
University of Oklahoma Health Sciences Center, news release June 10, 2010

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Comments

Thanks a Lot to all TEAM of Doctors, reseracher and others who help to find our soluation to RP , This is very good news in every one life that they can see again as well as those who has this type of problem. ONCe agaist THANK THANKS THANKS ALL THE TEAM
Please let me know how can i contact team of doctors as my brother has same disease RP. Please please let me know as early as possible.Thanks
My son Yashwant is started suffering from RP. He is of age 17 years. Please guide us or give your suggestion to make use of above gene therapy to my son. We would like to try this. By God grace, we can able to spend some money after we know about the details. Some of our relatives also staying in US, so that any physical help can be extended from them also. We will be grateful to you for this help. Thanks. S.Manikumar Reddy
Thank you for writing. I am not a medical professional--I have simply offered information about one area of progress in the field of retinitis pigmentosa. With the help of medical professionals, it may be helpful if you stay abreast of current research via clinical studies being conducted in various centers around the world as well as to contact organizations such as the American Academy of Ophthalmology, National Eye Institute, the National Organization for Rare Disorders, RP Fighting Blindness, and the latest research studies via PubMed. The best of luck to you.
my son is suffering from retinitis pegmentosa.his age is 16.I visits usa with my son and gave blood simpls in hope i will get breakthrough
MY SON AGE 16 IA SUFFERING FROM RETINITS PEGMENTOSA.I AM FROM INDIA. I ALREADY VISITED usa.I HAVE GIVEN OUR BLOOD SAMPLS IN SANFRASISCO.PLEASE HELP ME
I have retinitis pigmentosa,please i want to know when the medicine will be ready for this case... please reply for any information.... thank you
my son age 16 suffrring from retinits pegmentosa.after seeing this page.i hope somthing will come out soon .
I would like to know if you discovered a treatment for retinitis-pigmentosa and appreciate giving me some details on the treatments, the time needed and approximate cost thanks
kindly inform me whether treatment is available for rp or it is in testing stage
i had retinitis pigmentosa
how did you cure it?????????????????????
This article is about a gene therapy technique that may possibly prevent or cure retinitis pigmentosa. It is still under development. If you want more information about this technique, I suggest you contact the investigators at the University of Oklahoma Health Sciences Center or at the Dean McGee Eye Institute. Perhaps they can tell you about any clinical trials. Good luck.
JUST WANT TO KNOW IS THIS JUST A TRIAL OR A PROPER TREATMENT.
When this Gene therapy will be started. please advise thanks Kume
How can someone find out about clinical trials
My husband has retinitis pigmentosa. Can anybody help us can he be treath. Please contact us
my all three kids suffering from retintis pigmentosa, How much success expects in Stem Cell or Gene Therapy. Where it can be done and how much cost in US dollarls, what is procedure
Hi my younger brother aged 10 has retinitis Pigmentosa, doctors have said that he will be legally blind by the age of 30, i read with interest about this treatment. Is it still in studying stage or is it available for treatment now.... Please replyy Thank you...
The treatment is still being developed and is not available now.
my son is having RP & was diagonised at the age of six in the year 1993. Till date ,we are praying for solution to his problems. Otherwise he is quite brilliant in his studies and has completed his post graduation in Ist division. I am curious to know the cost of treatment & when this treatment would be commercially available. Kindly reply at the earliest. Thanks Mohinder Singh
I do not know the answers to your questions. However, you might want to contact the principal investigators of the study, including Dr. Muna Naash, at the University of Oklahoma. He or others at the University may be able to help you and also know of clinical trials utilizing this technique. Good luck to your son. Deborah Mitchell
i m 37 have same problem sir.
i'm 24 years old and i was diagnosed with retinitis pigmentosa at the age of 12.. in october of 2009 i was pronouced legally blind and was put on disabillity... i really want to find a way to have the rest of my sight back or prevent this from progressing any further... thank you much, Beckie Bonnewell
Beckie: The gene therapy discussed in this article is still in the development stages. One suggestion is to contact the principal investigators of the study at the Univ. of Oklahoma and see if there are any clinical trials or other avenues you might pursue. One promising thing is that research is progressing in this area. I wish you the best. Deborah Mitchell
hi, my son who is 4yrs old has been diagonised by RP... JUST AFEW DAYS AGO...... after doing his full field ERG TESTon both the eyes as per the INTERNATIONAL SOCIETY FOR CLINICAL ELECTROPHSSIOLOGY OF VISION (ISCEV) THE ELECTROPHYSIOLOGICAL IMPRESSION SAYS AS FOLLOWS... SEVERE ROD-CONE DYSFUNCTION IN BOTH EYES...AT THIS STAGE CAN HE B PREVENTED FROM TOTAL BLINDNESS...BY TAKING THE TREATMENT GENE THERAPY... PLS ADVICE.....UR REPLY VUD REALLY BRING US SOME HOPE'S IN OUR LIVES... REGARDS
I am sorry to hear about your son. In addition to the research mentioned in this study, other emerging technology involves an artificial retina, a topic I will cover in an article tomorrow. However, it is only approved in Europe at this time and is very expensive. Regards, Deborah Mitchell
My son 11 yrs old has retinitis pigmentosa, detected at 5yrs. We are from india, do u see any treatment in near future around the world.
I wish the best for your son. The treatment mentioned in this study is still experimental, however it is a good idea to keep looking for clinical trials anywhere in the world for which your son may qualify. Research is ongoing using gene therapy and other methods, so there is always hope.
Thank you very much for your reply, we really get hopes from it. Pls keep my email id in your record, if any treatment is available in the future pls update about it. Thanks & Regards.

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