Navigation Links
Therapy Yields Promise for Fatal Neurological Condition
Date:5/13/2008

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:
*Name:
*Comment:
*Email:
Related Image:
Therapy Yields Promise for Fatal Neurological Condition
(Date:3/24/2017)... ... March 24, 2017 , ... According to a new study by NCPA ... does not obey the rules Congress has directed the CBO to follow. The CBO ... reform would restore. Yet, it estimates a reduction in employer-based coverage due to the ...
(Date:3/24/2017)... Diego, Calif. (PRWEB) , ... March 24, 2017 ... ... has raised over $100,000 for its innovative EcoQube Frame vertical micro-veggies garden on ... the instant demand for the product – with nearly 2,000 consumers (and counting) ...
(Date:3/24/2017)... ... 2017 , ... Viewers who like to educate themselves on current issues and ... services, and societal issues tend to appreciate and love the "Informed" series, hosted by ... running events for causes around the world. , Running for charity has ...
(Date:3/24/2017)... ... March 24, 2017 , ... “End Time GPS”: ... events will interrelate. “End Time GPS” is the creation of published author, Wesley ... while working on military munitions and space-vehicle projects. Now, at age ninety-one, he shares ...
(Date:3/23/2017)... ... March 23, 2017 , ... A recent report from the National Council ... programs. The NCTQ report suggests, based on a review of GPA and SAT/ACT requirements ... quality in the U.S. It argues that this higher bar should be set by ...
Breaking Medicine News(10 mins):
(Date:3/24/2017)... , March 24, 2017 ... providing high-quality and cost-effective drug development and ... pharmaceutical and biotechnology industry, announced today the ... ShangPharma will be consolidating the Contract ... (CMO) under Shanghai ChemPartner. These entities include ...
(Date:3/24/2017)... Research and Markets has announced the addition ... - 2035" report to their offering. ... The Deep Learning: Drug Discovery and Diagnostics ... the growing market of deep learning solutions within the healthcare domain. ... emerged as a novel solution to generate relevant insights from medical ...
(Date:3/24/2017)... Research and Markets has announced the addition of the ... their offering. ... provides separate comprehensive analytics for the US, Canada , ... Asia-Pacific , Latin America , and Rest of ... 2022. Also, a six-year historic analysis is provided for these markets. Market ...
Breaking Medicine Technology: