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:7/30/2014)... WA (PRWEB) July 30, 2014 According to ... Vkool.com, this is a comprehensive guide that teaches people how ... few weeks. The book uncovers to readers the horrible truth ... in its review that inside this book, people will learn ... such as inflammation, asthma, and nasal infections. , Nasal Polyps ...
(Date:7/30/2014)... Kennewick, WA (PRWEB) July 30, 2014 ... today availability of over 80 different electronic therapy ... notes on tablets and other computing devices all ... 80+ therapy notes or electronic medial records (EMR) ... initial evaluations, and other essential categories. SOAP ...
(Date:7/30/2014)... Pain Free Living web: Superior Magnetics ... relief and improved performance. This new magnetic sports bracelet ... silicone core that is sheathed in nylon soaked in ... with scalar energy and Schumann frequency thus making it ... 12 years experience developing magnetic bracelets. We developed the ...
(Date:7/29/2014)... 2014 VisitandCare.com today announced ... added to the company’s exclusive network to help couples ... joint efforts — as top fertility clinics in Spain, ... affordable, state-of-the-art, and successful IVF treatments. , The exclusive ... comprehensive study by professionals at VisitandCare.com. The goal is ...
(Date:7/29/2014)... 30, 2014 Saint Luke’s Medical ... awarded the Patient-Centered Medical Home 2011 Level 3 ... for Quality Assurance. Only one out of four ... Level 2, or Level 3 recognition. , Patients ... Medical Home report faster access to their doctors, ...
Breaking Medicine News(10 mins):Health News:Nasal Polyps Treatment Miracle Book Review Exposes Manuel Richards' Guide For Treating Nasal Polyps – Vkool.com 2Health News:Nasal Polyps Treatment Miracle Book Review Exposes Manuel Richards' Guide For Treating Nasal Polyps – Vkool.com 3Health News:My Clients Plus Announces Over 80 Online Therapy Notes to Help Healthcare Providers Go Paperless 2Health News:Superior Magnetics Released New Magnetic Sports Bracelet for Pain 2Health News:Top European Fertility Clinics Join VisitandCare.com 2Health News:Top European Fertility Clinics Join VisitandCare.com 3Health News:Top European Fertility Clinics Join VisitandCare.com 4Health News:Saint Luke’s Medical Group Awarded Patient-Centered Medical Home 2011 Level 3 Recognition 2
... COSTA MESA, Calif., March 2 The deadliest wildfires ... 200 people and injured many more. Local medical institutions ... many with severe burns. The United States, largest tissue ... this crisis by providing skin to treat some of ...
... reason to eat your broccoli: UCLA researchers report that ... cruciferous vegetables may help protect against respiratory inflammation that ... pulmonary disease. , Published in the ... the research shows that sulforaphane, a chemical in broccoli, ...
... ARBOR, Mich., March 2 NanoBio Corp. announced today ... clinical and preclinical milestones in the development of its ... capitis. The new data will be delivered in five ... of Dermatology in San Francisco, March 6-9, 2009. In ...
... likely driven by limited availability of adolescent-only services , , ... percent of the 1.4 million American teens with substance ... study. , "Part of this treatment gap may ... services. Less than one-third of addiction programs in the ...
... more than half of MS patients are overweight or obeseWOONSOCKET, ... CVS ) today said it is working to help ... that found more than half of individuals with MS can ... recent review of CVS Caremark health management clients, plan participants ...
... The Healthcare business of Thomson Reuters has been ... its preferred vendor for mobile clinical quality improvement solutions. ... authorized to sell its Clinical Xpert(TM) suite of products ... the KLAS Category Leader for Mobile Data Systems for ...
Cached Medicine News:Health News:MTF, Largest Tissue Bank, Provides Skin for Victims of Australian Wildfires 2Health News:Broccoli may help protect against respiratory conditions like asthma 2Health News:NanoBio to Announce Key Clinical and Preclinical Data on Its Topical Anti-infective Lotions at AAD 2009 2Health News:Teens Undertreated for Substance Abuse 2Health News:CVS Caremark Health Management Programs Help Multiple Sclerosis Patients Better Manage Weight Issues and Improve BMI 2Health News:CVS Caremark Health Management Programs Help Multiple Sclerosis Patients Better Manage Weight Issues and Improve BMI 3Health News:VHA Selects Clinical Xpert from Thomson Reuters as its Preferred Solution for Mobile Clinical Improvement Systems 2Health News:VHA Selects Clinical Xpert from Thomson Reuters as its Preferred Solution for Mobile Clinical Improvement Systems 3
(Date:7/29/2014)... Laboratory (IL) today announced the launch of its new corporate ... of a comprehensive rebranding effort by Werfen, the parent company ... , in 1959, IL has been part of the ... the group include Inova Diagnostics ( San Diego, CA ... ).  Like IL, these three companies are focused on ...
(Date:7/29/2014)... , July 29, 2014 Research and ... "Global Respiratory Devices Market 2014-2018" report to their ... A respiratory device is a medical device used ... having difficulty in breathing and cannot achieve adequate oxygen ... as cystic fibrosis, asthma, COPD, and ARDS require the ...
(Date:7/29/2014)... , July 29, 2014  Based on its ... (PERS) market, Frost & Sullivan recognizes MobileHelp with ... for Price Performance Value Leadership. MobileHelp,s leadership in ... expectations for tech progression in personal emergency response, ... these solutions. MobileHelp is at the ...
Breaking Medicine Technology:Instrumentation Laboratory Unveils New Corporate Brand 2Instrumentation Laboratory Unveils New Corporate Brand 3Global Respiratory Devices Market 2014-2018 2Frost & Sullivan Applauds MobileHelp for its Targeted Supplies for the PERS Market 2Frost & Sullivan Applauds MobileHelp for its Targeted Supplies for the PERS Market 3
... Injection is supplied in an isotonic solution ... intravenous administration. Each milliliter contains 37 megabecquerels ... calibration time, made isotonic with 9 milligrams ... benzyl alcohol. The pH is adjusted to ...
... single-detector system offers general purpose ... for gurneys and wheelchairs. This ... versatile open gantry, caudal/cephalic detector ... for SPECT and whole body ...
Stainless steel lancet blade in stainless steel....
Sharp diamond shaped double blade in stainless steel....
Medicine Products: