Navigation Links
Gene Therapy Offers Hope for Severe Hereditary Eye Disease
Date:10/24/2009

Youngest patients have best chance at some visual recovery, research suggests

SATURDAY, Oct. 24 (HealthDay News) -- Gene therapy can improve the vision of people with a severe form of genetically inherited retinal degeneration called Leber congenital amaurosis (LCA), a new study shows.

People with LCA, which is caused by mutations in any of least 13 genes, have severe vision loss and abnormal eye movements in early infancy and during early childhood. Most people with the disease are blind by their 30s or 40s. There is no cure, according to background information provided in a news release.

The new phase I trial included 12 patients, aged 8 to 44 years, who were injected in one eye with genetic material meant to correct LCA. The gene therapy led to at least a 100-fold increase in pupillary light response (constriction of the pupil when it's exposed to light) in the participants. An 8-year-old patient developed nearly the same level of light sensitivity as a person with normal vision, the study authors reported.

In general, the greatest improvements were seen in children aged 8 to 11, all of whom gained ambulatory vision -- being able to see well enough to walk unaided.

"All 12 patients given gene therapy in one eye showed improvement in retinal function. The effect was stable during follow-up. The results support our hypothesis that the response to subretinal gene therapy depends on the extent of retinal degeneration and, therefore, the age of the patient," wrote Dr. Jean Bennett, of the University of Pennsylvania School of Medicine in Philadelphia, and colleagues.

"The most noteworthy result was the ability of children to navigate an obstacle course independently and accurately, even in dim light," the study authors wrote. "The visual recovery noted in the children confirms the hypothesis that efficacy will be improved if treatment is applied before retinal degeneration has progressed. Assessment of whether the treatment alters the natural progression of the retinal degeneration will be possible in follow-up studies," they added.

"The success of this gene therapy study in children provides the foundation for gene therapy approaches to the treatment of other forms of LCA and of additional early onset retinal diseases," the researchers concluded.

The study is published in the Oct. 24 online edition and in an upcoming print issue of The Lancet.

More information

The U.S. National Eye Institute has more about gene therapy for LCA.



-- Robert Preidt



SOURCE: The Lancet, news release, Oct. 24, 2009


'/>"/>
Copyright©2009 ScoutNews,LLC.
All rights reserved

Related medicine news :

1. Vision restoration therapy shown to improve brain activity in brain injured patients
2. HIV therapy in pregnancy-data support WHO recommendations
3. Family-based treatment more effective than supportive psychotherapy in treating bulimia
4. Trial to Test Gene Therapy for Angina in Women
5. One of the Largest Post-WHI Physician Surveys Shows More Education is Needed: Patient Misinformation About Hormone Therapy Remains High
6. Aromatherapy Gift Line Sheds Humorous Light on Modern Therapy
7. Chemotherapy may be culprit for fatigue in breast cancer survivors
8. Atlanta Falcons Physical Therapy Centers Set to Launch This Fall
9. New Drug No Substitute for Standard Blood-Clot Therapy
10. Stem Cell Therapy Disappoints Against Rare Kidney Ailment
11. Nucletron Announces Management Buy-Out to More Effectively Meet the Demands of Its Customers and the Radiation Therapy Community
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/21/2017)... ... July 21, 2017 , ... ... financial consultation services to families and business owners in the Dalton region, is ... support for children overcoming sexual assault and physical abuse. , The GreenHouse is ...
(Date:7/21/2017)... ... July 21, 2017 , ... ... and financial planning services to families and business owners in northern Florida, is ... promises to provide support to area adults with developmental challenges. , The First ...
(Date:7/21/2017)... ... July 21, 2017 , ... Fresh Wave® IAQ today announced the ... solution for colleges and universities at the APPA 2017 Annual Conference and ... and tobacco smoke odors without the use of harsh chemicals, Fresh Wave IAQ Smoke ...
(Date:7/21/2017)... ... July 21, 2017 , ... Bernard R. Bach, Jr., ... of the American Orthopaedic Society of Sports Medicine (AOSSM) , received the 2017 ... AOSSM Annual Meeting in Toronto, Canada. This prestigious award is given annually to honor ...
(Date:7/21/2017)... , ... July 21, 2017 , ... ... received a $5,000 grant from the C. R. Bard Foundation, Inc. ... at Somerset Hills , a service available through the nonprofit home care agency. ...
Breaking Medicine News(10 mins):
(Date:7/5/2017)... July 5, 2017 Pace Analytical, a company of over 2,000 employees ... they have acquired ESC Lab Sciences, further solidifying their position as the top ... . ... President and CEO of Pace Analytical ... Sciences out of Mt Juliet, TN , enhances Pace Analytical,s ...
(Date:7/1/2017)... , June 30, 2017 Zimmer Biomet Holdings, Inc. ... and earnings conference call will be broadcast live over the ... Time.  A news release detailing the quarterly results will be ... the conference call. The live audio webcast can ... http://investor.zimmerbiomet.com . It will be archived for replay following the ...
(Date:6/30/2017)... 2017  AVACEN Medical (AVACEN) announced the publication of new research ... use of its AVACEN Treatment Method to significantly reduce ... ... Medical ... It affects approximately 200 to 400 million people worldwide according to ...
Breaking Medicine Technology: