Navigation Links
Gene Therapy Shows Promise for Rare Children's Brain Disorder
Date:12/19/2012

By Amy Norton
HealthDay Reporter

WEDNESDAY, Dec. 19 (HealthDay News) -- Gene therapy could offer a safe option for treating children with a rare degenerative brain disease, a preliminary study suggests.

The condition, known as Canavan disease, is caused by a mutation in the ASPA gene, which codes for an enzyme called aspartoacylase. Without that enzyme, a chemical known as N-acetylaspartic acid (NAA) builds up in the brain and prevents nerve cells from developing their normal protective sheath. Eventually, the brain degenerates into spongy tissue with fluid-filled spaces.

Canavan disease is rare, with an estimated 600 cases worldwide, according to Paola Leone, the lead researcher on the new study and an associate professor of cell biology at the University of Medicine and Dentistry of New Jersey in Stratford, N.J.

Both parents have to carry the mutated gene for a child to be affected, and the disease is most common among people of Ashkenazi Jewish heritage.

Babies born with Canavan disease never develop normal movement and thinking abilities. They have weak and stiff muscles, suffer seizures, and have problems eating and even supporting their head. (The disorder causes the head to become unusually large.)

Some children with Canavan disease live into their teens or 20s, but most die before their 10th birthday.

"Fifteen years ago, there was no hope for these children," Leone said.

Back in 2001, though, U.S. regulators gave researchers the go-ahead to study a gene therapy method developed by scientists at the University of North Carolina School of Medicine.

The tactic involved inserting a normal copy of the ASPA gene into a non-infectious virus. That gene-carrying virus, or vector, was then infused directly into the brains of children with Canavan disease, in an attempt to override the defective gene.

Over the course of a few years, the researchers treated 13 children in all, and
'/>"/>

Copyright©2012 ScoutNews,LLC.
All rights reserved  

Page: 1 2 3 4

Related medicine news :

1. People with HIV hospitalized less often since combination antiretroviral drug therapy introduced
2. Shaft Medical San Diego now offers micro needle sclerotherapy to treat scary “spider” veins.
3. Churchview Supportive Living to Host Music Therapy Informational Program
4. UAlberta medical researchers discover new potential chemotherapy
5. New hormone therapy shows promise for menopausal symptoms in animal model
6. Older and younger chronic leukemia patients may need different therapy
7. Novel test identifies patients most likely to benefit from ALK inhibition therapy
8. Combo Therapy May Help Ease Sleep Apnea at High Altitude
9. Potential gene therapy approach to sickle cell disease highlighted at American Society of Hematology
10. Dr. Farshchian: Labrum Tear is Now Being Treated With Cell Therapy at the Center for Regenerative Medicine Using
11. Moffitt researchers say effective immunotherapy for melanoma hinges on blocking suppressive factors
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Gene Therapy Shows Promise for Rare Children's Brain Disorder
(Date:7/5/2015)... ... , ... City Sports, the Boston-based specialty sporting goods retailer, has renewed their ... triathletes this summer with ethos, the owner and operator of the Boston Triathlon. , ... p.m. at the North End Greenway. The event will help new triathletes prepare for ...
(Date:7/5/2015)... PA (PRWEB) , ... July 05, 2015 , ... ... of patented products, announces the Weed Master, a landscaping invention that effectively ... worth $75 billion US dollars," says Scott Cooper, CEO and Creative Director of ...
(Date:7/5/2015)... ... July 05, 2015 , ... Doctors at the Swedish ... IMRT/surgery combination may one day change the outlook for people with pleural mesothelioma. Surviving ... , Researchers report that five mesothelioma patients treated with 25Gy of radiation ...
(Date:7/4/2015)... ... July 04, 2015 , ... ... published the results of an annual member survey , detailing the most ... More recently, the group broke that raw data down into charts detailing demographic ...
(Date:7/4/2015)... York, NY (PRWEB) , ... July 04, 2015 , ... When a hacker gains access ... home front and at the office. On Wednesday, July 1st, the FTC issued a ... credentials as part of a verification process for a password reset instigated by the hacker. ...
Breaking Medicine News(10 mins):Health News:Sporting Goods Retailer City Sports Renews Partnership with Boston Triathlon 2Health News:Sporting Goods Retailer City Sports Renews Partnership with Boston Triathlon 3Health News:8 Landscaping Tips That Can Save You Money Thanks To World Patent Marketing's Innovative Landscaping Invention 2Health News:8 Landscaping Tips That Can Save You Money Thanks To World Patent Marketing's Innovative Landscaping Invention 3Health News:8 Landscaping Tips That Can Save You Money Thanks To World Patent Marketing's Innovative Landscaping Invention 4Health News:8 Landscaping Tips That Can Save You Money Thanks To World Patent Marketing's Innovative Landscaping Invention 5Health News:New Mesothelioma Study Calls IMRT/Surgery Combo “Innovative and Promising”, According to Surviving Mesothelioma 2Health News:The ‘South Atlantic’ Frequently Chooses Tummy Tuck for Plastic Surgery, Jacksonville, FL, Doctor Notes 2Health News:The ‘South Atlantic’ Frequently Chooses Tummy Tuck for Plastic Surgery, Jacksonville, FL, Doctor Notes 3Health News:New FTC Scam Alert Shows Latest Example of Social Engineering Dangers 2Health News:New FTC Scam Alert Shows Latest Example of Social Engineering Dangers 3Health News:New FTC Scam Alert Shows Latest Example of Social Engineering Dangers 4
... COLUMBIA, Mo. Despite billions of dollars spent on ... out of 33 developed countries for life expectancy at ... to pre-term birth or low birth weight-outcomes that have ... researcher says a key factor in addressing this issue ...
... world is a dazzling array of people, objects, sounds, smells ... at any moment. So our attention may automatically be snagged ... may deliberately focus on something that is important to us ... screaming hordes on the school playground. We also know that ...
... 7 (HealthDay News) -- Women who gain an excessive amount ... possible risk factor for childhood obesity, new research suggests. ... 25 to 35 pounds during pregnancy. Overweight women should keep ... should limit weight gain to 11 to 20 pounds. ...
... In what is believed to be the first published ... University School of Medicine (BUSM) believe female military service-members ... be as resilient to combat-related stress as men. These ... Abnormal Psychology . Though the literature suggests ...
... people out of the criminal justice system and into mental ... research from Queen Mary, University of London. In a study ... , researchers estimate that there are over 8,000 prisoners with ... If transferred to hospital, treatment for these patients would have ...
... fellow in cardiothoracic surgery research at Rhode Island Hospital, ... Surgery Foundation for Research and Education (TSFRE) to support ... Model of Chronic Cardiac Ischemia." TSFRE awarded just two ... years. TSFRE fellowships are designed to provide salary ...
Cached Medicine News:Health News:Health care providers need training to recognize signs of domestic violence, says nursing expert 2Health News:Your attention please: 'Rewarding' objects can't be ignored 2Health News:Your attention please: 'Rewarding' objects can't be ignored 3Health News:Too Much Weight Gain in Pregnancy Linked to Chubby Babies 2Health News:First of its kind study conducted by BUSM finds women as resilient to combat stress as men 2Health News:Rhode Island Hospital fellow receives funding to support research in myocardial perfusion 2
(Date:7/3/2015)... , Mexique, July 3, 2015 ... continue cible les épidémies que sont l,hypertension, le ... par l,intermédiaire de plateformes éducatives gratuites et d,une ...      (Photo: http://photos.prnewswire.com/prnh/20150630/227033 ) , ... diabète en ligne , Prendre en charge ...
(Date:7/3/2015)... , July 3, 2015 ... with Broda Technologies Co., Ltd., operating in both the ... the company,s proprietary IntelliGel ® supramolecular hydrogel technology ... development and other ophthalmic therapies. The Broda technology offers ... solution is a free-forming liquid and with low concentrations ...
(Date:7/2/2015)... and VANCOUVER , July 2, 2015 ... EPI) announced today that its common shares have been approved ... under the symbol "EPIX". ESSA will begin trading ... its listing on the TSX Venture Exchange under the symbol ... the common shares on the NASDAQ, each of the Company,s ...
Breaking Medicine Technology:Lutter contre trois maladies chroniques majeures en Amérique latine grâce à la formation médicale 2Lutter contre trois maladies chroniques majeures en Amérique latine grâce à la formation médicale 3Redwood Pharma Licenses Novel Hydrogel Drug Delivery Technology From Broda Technologies 2ESSA Pharma Inc. to Commence Trading on the NASDAQ Capital Market 2ESSA Pharma Inc. to Commence Trading on the NASDAQ Capital Market 3ESSA Pharma Inc. to Commence Trading on the NASDAQ Capital Market 4
... Diabecline® antibiotic ointment (SSPT® Tetracycline) received the Bronze Award ... Annual Edison Awards in New York City.  pharmaCline joined ... the New York Academy of Sciences, which hosted the ... Gala event acknowledged the hard work, innovation and commitment ...
...  Cohera Medical, Inc.®, a leading innovator and developer of ... of the first patient in a prospective, multicenter, randomized ... Surgical Adhesive in the United States.  Initiation of the ... of the lead product toward commercialization in the United ...
Cached Medicine Technology:Diabecline Antibiotic Receives Edison Award for Best New Pharmaceutical Product 2Diabecline Antibiotic Receives Edison Award for Best New Pharmaceutical Product 3First Patient Treated in U.S. Clinical Study of Cohera Medical's TissuGlu® Surgical Adhesive 2First Patient Treated in U.S. Clinical Study of Cohera Medical's TissuGlu® Surgical Adhesive 3
The K-ASSAY Rheumatoid Factor Control Set is intended to be used as a consistent test sample of known concentration for monitoring the performance of K-ASSAY immunoturbidimetric assay for quantifying...
... The K-ASSAY Microalbumin Urine Controls ... to use liquid requiring no ... prepared from human urine, fortified ... albumin and creatinine. Preservatives including ...
... Liquid Lipid Control is intended for use ... test sample of known concentration for monitoring ... K-ASSAY Liquid Lipid Control with patient serum ... listed constituents. The user can compare observations ...
The K-ASSAY ITA Lyophilized Multi-Control Set is intended to be used as a consistent test sample of known concentration for monitoring the performance of K-ASSAY immunoturbidimetric assays for the li...
Medicine Products: