Navigation Links
Safety study indicates gene therapy for blindness improves vision
Date:9/8/2008

GAINESVILLE, Fla. All three people who received gene therapy at the University of Florida to treat a rare, incurable form of blindness have regained some of their vision, according to a paper published online today in Human Gene Therapy.

The patients one woman and two men ranging from 21 to 24 years old with a type of hereditary blindness called Leber congenital amaurosis type 2 volunteered to test the safety of an experimental gene-transfer technique in a phase 1 clinical research study conducted by UF and the University of Pennsylvania with support from the National Eye Institute of the National Institutes of Health.

In this form of LCA disease, photoreceptor cells cannot respond to light because a gene called RPE65 does not properly produce a protein necessary for healthy vision. In the study, researchers used an adeno-associated virus an apparently harmless virus that already exists in most people to deliver RPE65 to a small area of the retina.

Not only were there no ill effects other than routine postsurgical soreness, the subjects said the vision in their treated eyes was slightly improved in dim lighting conditions.

"The patients report seeing brighter areas and perhaps some images, but basically the message is that this is treatment is fully safe," said William W. Hauswirth, Ph.D., a professor of ophthalmology and member of UF's Powell Gene Therapy Center and the UF Genetics Institute.

"One thing we did not do is suppress the patients' immune systems, which was done in two other LCA clinical trials that were under way," said Hauswirth, who began studying the adeno-associated virus as a vehicle to deliver genes into living animals more than 30 years ago. "Theoretically, the idea was that it might be necessary to suppress the immune system because we are using a vector that might activate the body's defenses and cause a harmful response. However, immune suppression itself carries a risk of infections and other problems. Clearly we have shown there is no need to do that in this case."

Samuel G. Jacobson, M.D., Ph.D., a professor of ophthalmology with the Scheie Eye Institute at the University of Pennsylvania, is the study's principal investigator.

"This groundbreaking gene therapy trial builds on 15 years of research sponsored by the National Eye Institute of NIH," said Paul A. Sieving, M.D., Ph.D., director of the NEI. "The study has partially restored vision in three young adults, and it demonstrates that gene therapy can be effective in treating human vision disease. Many human diseases are inherited in families and result from mutations in single genes. These genetic conditions are particularly suited to potential treatment by gene therapy. This trial to treat vision loss from the condition of Leber congenital amaurosis is an important demonstration of proof of principle and shows that we are on the right track. We can now invest in further work to refine, and ultimately to expand, genetic treatment approaches."

Results published today focus on the health of the entire retina, not just the tiny portion that received the gene therapy. A detailed examination of the therapy's effectiveness in the treated portion of the eye will appear in an upcoming issue of the Proceedings of the National Academy of Sciences. Two other recent LCA clinical trial reports appeared recently in The New England Journal of Medicine.

"The safety study itself is a milestone, but when we see a benefit to the subject that is a truly a welcome bonus," said Barry J. Byrne, M.D., Ph.D., a professor of molecular genetics and microbiology and director of UF's Powell Gene Therapy Center, which manufactured the viral vectors used in the study. "Improvements in someone's medical condition are ultimately what we are after."

LCA2 affects about 2,000 people in the United States and is one of several incurable forms of blindness collectively known as retinitis pigmentosa, which in turn affects about 200,000 Americans.

Children with LCA2 experience major visual disability that can lead to total vision loss in adulthood. Although vision loss is severe, the structure of the retina including its connection to the brain can remain relatively intact for decades before the photoreceptor cells degenerate.


'/>"/>

Contact: John Pastor
jdpastor@ufl.edu
352-273-5815
University of Florida
Source:Eurekalert

Related medicine news :

1. More proof needed of safety and quality of electronic personal health records
2. Back to School Means Backpack Safety
3. Seniors Need a Safety Plan in Face of Disasters
4. New Survey Shows Americans are Still Concerned About Food Safety, Yet Still Not Smart About What They Like to Eat
5. National Patient Safety Foundation Partners with Vocera Communications
6. QI projects may -- or may not -- improve patient safety and outcomes
7. The National Center for Missing & Exploited Children, Wal-Mart, and Abbott Host Child Safety Events
8. Cutting Salt Wont Affect Foods Safety
9. Microsoft Helps Developers of Clinical User Interfaces Improve Efficiency, Patient Safety With Free Toolkit
10. The Risk Management and Patient Safety Institute Announces Annual Health Care Risk Conference Dates
11. Blue Cross and Blue Shield Initiatives Recognized for Increasing Patient Safety and Efficiency
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/26/2017)... ... , ... “When the Stars Lead Home”: a poignant story of loss, determination, ... Weigel Douglas, an avid reader who lives in the Pacific Northwest with her husband, ... Hills Adventure Camp. She couldn’t be more grateful. , Twelve-year old Tizzy could not ...
(Date:5/26/2017)... ... May 26, 2017 , ... ... recipient of proceeds from its 14th Annual Clays for Kids fundraiser, to be ... 30, Bennett, Colorado. , As part of BluSky’s partnership with The Adoption ...
(Date:5/26/2017)... ... ... “Cactus Jack: Against All Odds”: the story of Coach Cactus Jack and ... Odds” is the creation of published author, Walter Hubbard, a retired wildlife and fisheries ... child Jane. Walter. Walter and Jane have three adult children and a granddaughter. ...
(Date:5/24/2017)... BELLEVILLE, N.J. (PRWEB) , ... May 24, 2017 ... ... joined Clara Maass Medical Center CEO Mary Ellen, hospital employees, and town officials ... in Belleville. , The facility was developed by Rendina as part of its ...
(Date:5/24/2017)... ... May 24, 2017 , ... Dr. Alan I. Benvenisty, MD ... New York City. He is known for his distinguished expertise and experience in the ... Benvenisty holds sub-specialty training in treating renovascular disease and aortic aneurysm . He ...
Breaking Medicine News(10 mins):
(Date:5/26/2017)... , May 25, 2017  In response to ... , Direct Relief is working with Pfizer to ... available at no cost to community health centers, free ... providers nationwide. "Pfizer has a long-standing ... medicines and ensuring patient safety through educational activities," said ...
(Date:5/22/2017)... 2017  Lilac Corp, the company that sells ... of a new website . The website ... clinical study that showed surprising clearance of the ... individuals suffering from HPV warts, precancerous, or cancerous ... no other treatments that clear the virus. Specifically, ...
(Date:5/18/2017)... May 17, 2017  Bayer announced today that the ... be presented at the 53 rd Annual Meeting ... place June 2-6 in Chicago . ... span prostate, colorectal, liver and thyroid cancers, as well ... Phase II CHRONOS-1 trial of copanlisib in patients with ...
Breaking Medicine Technology: