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:1/19/2017)... Angeles (PRWEB) , ... January 19, 2017 , ... Next ... Act will be repealed by Congressional political games that circumvent health needs of over ... Stand Rally” capture the human anxieties and needs government public servants were suppose to ...
(Date:1/19/2017)... ... 2017 , ... WhoHaha , a digital media company dedicated to creating ... to produce a three-part video series that uses humor to highlight ways to improve ... of AHA’s Healthy For Good™ movement, which is designed to inspire all Americans to ...
(Date:1/19/2017)... ... January 19, 2017 , ... Dr. ... Board of Dermatology and fellowship trained Mohs and cosmetic surgeon. After extensive dermatology ... Dr. Li completed his internship in internal medicine at the Emory University and ...
(Date:1/19/2017)... ... January 19, 2017 , ... ... treatments for patients with sleep apnea and TMJ disorders. These conditions are quite ... health. Dr. Rassouli provides personalized care to reduce the risk of these consequences ...
(Date:1/19/2017)... ... January 19, 2017 , ... ... and financial planning services to communities throughout eastern Georgia, is embarking on a ... disease. , Heart disease kills more Americans every year than anything else, yet ...
Breaking Medicine News(10 mins):
(Date:1/19/2017)... This report on the opioid induced constipation ... the global market. Large number of chronic pain sufferers ... is a major side effect of consumption of opioid ... therapy has been prescribed to treat opioid induced constipation. ... and growing awareness about the therapy are the major ...
(Date:1/19/2017)... NEW YORK , January 19, 2017 ... global market for cryotherapy is set to witness a CAGR of ... North America will continue to be the leading ... ... Suppliers are emphasizing on ensuring affordable and adequate supply ...
(Date:1/19/2017)... 19, 2017  Sensus Healthcare, Inc. (NASDAQ: ... in the treatment of non-melanoma skin cancers and ... radiation therapy, today announced that it will report ... results on Thursday, February 2, 2017 after the market ... conference call with the investment community on Thursday, February ...
Breaking Medicine Technology: