Navigation Links
Therapy Yields Promise for Fatal Neurological Condition

Using harmless cold virus, injecting healthy gene into brain slowed Batten disease

TUESDAY, May 13 (HealthDay News) -- A new gene therapy that involves injecting a harmless virus into the brain shows promise as a safe and effective way to slow Batten disease, a rare neurological disorder that usually becomes fatal between the ages of 8 and 12.

Children with the disease, also known as Late Infantile Neuronal Ceroid Lipofuscinosis, are born with a mutated CLN2 gene. This causes a deficiency in TTP-1, an enzyme responsible for ridding cells of the central nervous system of waste materials. Parts of the neurons in the brain cell eventually become clogged with toxic material.

"It's like the garbage man of the cell is not able to do its job," study author Dr. Ronald Crystal, chairman of the Department of Genetic Medicine and chief of the Division of Pulmonary and Critical Care Medicine at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, said in a prepared statement. "The trash keeps getting backed up inside the cell until the cells can no longer function properly and then eventually die throughout the entire brain."

In a clinical trial, reported on in the May 13 online issue of Human Gene Therapy, neurological surgeons created six tiny holes in the skull of a patient, and then injected into the brain a liquid containing the healthy CLN2 gene within the harmless adeno-associated virus (AAV).

"The virus is used as a Trojan horse that houses and then delivers a healthy, functional gene into the cells of the brain," Crystal said. "The genes are incorporated within the genetic material of the cells, which are then able to produce a protein that is deficient in Batten disease."

In following the patients for 18 months after the procedure, the team found the gene therapy safely and effectively slowed the disease's progression.

"Before now, we had no hope of a therapy for Batten disease, but today, we can say that there is some hope," Crystal said. "These results are not just promising for sufferers of the disease, but suggest that gene therapy can work and should be studied for other neurological disorders. Each gene in our body has the potential to become a target to study for human disease."

Only about 200 cases of Batten disease exist in the world at a given time because of its early fatality rate. Around age 4, children with Batten disease usually start showing symptoms such as impaired muscle coordination (ataxia), involuntary twitching (myoclonus), and speech and developmental disorders. Eyesight then usually begins to decline, and affected children generally become wheelchair-bound, then bedridden.

More information

The National Institute of Neurological Disorders and Stroke has more about Batten disease.

-- Kevin McKeever

SOURCE: NewYork-Presbyterian Hospital/Weill Cornell Medical Center, news release, May 13, 2008

Copyright©2008 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:
Related Image:
Therapy Yields Promise for Fatal Neurological Condition
(Date:11/25/2015)... San Mateo, CA (PRWEB) , ... November 25, 2015 , ... ... $15,000 in prizes were awarded to winners of the Create Real Impact awards. ... and creative expression to help stem the tide of distracted and reckless driving, the ...
(Date:11/24/2015)... ... November 24, 2015 , ... Dr. Todd S. Afferica, a ... services to many of his patients. Dr. Afferica now uses the BIOLASE WaterLase iPlus ... amount of time the doctor uses other traditional cutting tools, such as the scalpel ...
(Date:11/24/2015)... , ... November 24, 2015 , ... ... launched their Black Friday sale a week early, offering 40% off select bras ... the intimate apparel industry through both mobile fit technology and the latest fashion, ...
(Date:11/24/2015)... IL (PRWEB) , ... November 24, 2015 , ... ... are national leaders when it comes to several aspects of orthopedic care. They ... joint replacements, orthopedic surgeries and general orthopedic care. , Becker's Hospital Review ...
(Date:11/24/2015)... ... November 24, 2015 , ... Brillouin Energy Corp. Congress ... the developer of renewable energy technologies capable of producing commercially useful amounts of ... that its WET™ and HHT™ Boiler System reactor core modules were presented to ...
Breaking Medicine News(10 mins):
(Date:11/25/2015)... , Nov. 25, 2015 /PRNewswire/ ... "Company") (NYSE: MR ), a ... medical devices worldwide, today announced that ... general meeting of shareholders at the ... Grand Century, 193 Prince Edward West Road, ...
(Date:11/25/2015)... Pennsylvania , November 25, 2015 - ... Continuing Medical Education (CME) --> ... Continuing Medical Education (CME) --> ... Care Continuing Medical Education (CME) ... medical information products and services, will feature latest diagnostic imaging textbooks ...
(Date:11/25/2015)... , Frankreich, November 25, 2015 ... heute bekanntgegeben, dass sie eine Lizenz für das Patent ... Poxel, CNRS, UCBL und ENS-Lyon innehaben, an Enyo Pharma ... von FUI AAP8 ins Leben gerufenen und von Edelris ... wurde FXR als ein Behandlungsziel für HBV identifiziert, und ...
Breaking Medicine Technology: