Navigation Links
Gene Therapy Slows Progression of Fatal Neurodegenerative Disease in Children
Date:5/13/2008

NEW ROCHELLE, N.Y., May 13 /PRNewswire/ -- Gene therapy to replace the faulty CLN2 gene, which causes a neurodegenerative disease that is fatal by age 8-12 years, was able to slow significantly the rate of neurologic decline in treated children, according to a paper published online ahead of print in the May 2008 issue (Vol. 19 No. 5) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The paper is available free online at http://www.liebertpub.com/hum

Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) is an autosomal recessive genetic disorder that causes degeneration of the central nervous system. It is a form of Batten disease, a group of lysosomal storage disease in which a lipofuscin-like material is not broken down and accumulates in neurons, causing cognitive impairment, visual failure, seizures, and progressive deterioration of motor function.

Ronald Crystal and colleagues from Weill Cornell Medical College (New York, NY), describe a study conducted in 10 children with LINCL who received gene therapy to replace the defective CLN2 gene via administration of human CLN2 carried in an adeno-associated virus (AAV). In the paper entitled "Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis with CNS Administration of a Serotype 2 Adeno-associated virus expressing the CLN2 cDNA," the authors report that over an 18-month period, assessment using a neurologic rating scale demonstrated significant slowing of disease progression in the treated, compared to the untreated children. On the basis of these findings, the authors proposed that additional studies to assess the safety and efficacy of AAV-mediated gene therapy for LINCL be pursued.

Although the treatment was associated with some serious adverse events in some patients, these were not unequivocally attributable to the gene therapy vector.

"This clinical trial is an important step to
'/>"/>

SOURCE Mary Ann Liebert, Inc.
Copyright©2008 PR Newswire.
All rights reserved

Page: 1 2

Related medicine technology :

1. Velcura Therapeutics, Inc. Reports Successful Phase 1 Clinical Trial of Novel Bone Disease Therapy
2. Skin Flaps Deliver Cancer-fighting Therapy, ASPS Study Reveals
3. New Data Show High Frequency of Medical Claims for Gastrointestinal Events by Patients on Antiplatelet Therapy
4. QRxPharma Releases Successful Phase 3 Study Results for Dual Opioid Pain Therapy
5. Studies Point to Benefits of Personalized Chemotherapy Dose Management in Colorectal Cancer
6. CureDMs Diabetes Therapy Achieves Major Development Milestone
7. Gene Therapy Improves Vision in Patients with Congenital Retinal Disease
8. Gene Therapy Provides Vision to People who Were Nearly Blind
9. Dr. Calvin Grant Presents Results from Xibrom(TM) Combination Therapy with Lucentis(R) at the 2008 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO)
10. FTY720, a novel oral therapy in development for MS, shows sustained benefits for the majority of patients after three years of treatment
11. In Blood Vessel Stents, Innovative Materials Allow Better Control, Delivery of Gene Therapy
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/15/2014)... AARP Foundation today announced it has established a relief ... cold weather that has gripped much of the nation. AARP ... so to support these emergency relief efforts AARP and AARP ... mean up to $500,000 in aid. The matching program will ...
(Date:1/15/2014)... , Jan. 15, 2014  Celsion Corporation (the "Company") (NASDAQ: ... commitments from institutional investors to purchase an aggregate of ... an at-the-market registered direct offering, led by a dedicated ... definitive purchase agreements with these investors pursuant to which ...
(Date:1/15/2014)... , January 15, 2014 Shire plc (LSE: SHP, ... Office of Fair Trading (OFT) approval condition to the announced tender ... VPHM).   As a result of the waiver, the ... to complete the tender offer on January 24, 2014 following the ...
Breaking Medicine Technology:AARP Foundation Establishes Winter Relief Fund for Older Adults Suffering from the Severe Cold Weather 2AARP Foundation Establishes Winter Relief Fund for Older Adults Suffering from the Severe Cold Weather 3Celsion Corporation Announces $15 Million At-The-Market Registered Direct Offering 2Celsion Corporation Announces $15 Million At-The-Market Registered Direct Offering 3Shire Waives OFT Condition Relating to the Acquisition of ViroPharma Inc. 2Shire Waives OFT Condition Relating to the Acquisition of ViroPharma Inc. 3
... 16, 2011 /PRNewswire-Asia-FirstCall/ -- China ... (Nasdaq: CMED ), ... diagnostic ("IVD") company, today announced ... at the following investor conferences ...
... artery disease (CAD), also known as coronary heart disease, ... affecting about 17 million Americans(1). Most of these people ... angina pectoris. For over 100 years, physicians have treated ... nitroglycerin(2). Considered the gold standard in treating acute angina, ...
Cached Medicine Technology:China Medical Technologies to Meet Investors 2Akrimax Pharmaceuticals, LLC, Introduces NitroMist® (Nitroglycerin Lingual Aerosol) 400mcg/Spray 2Akrimax Pharmaceuticals, LLC, Introduces NitroMist® (Nitroglycerin Lingual Aerosol) 400mcg/Spray 3Akrimax Pharmaceuticals, LLC, Introduces NitroMist® (Nitroglycerin Lingual Aerosol) 400mcg/Spray 4
(Date:7/9/2014)... injection drug users in Russia might contribute to ... study, conducted by researchers from Boston University Schools ... St. Petersburg Pavlov State University, sought to discover ... outcomes of a cohort of HIV-positive people with ... were arrested by police were more likely to ...
(Date:7/9/2014)... YORK (July 8, 2014) Researchers from Montefiore ... of Medicine of Yeshiva University will present new ... held July 12 July 17 in Copenhagen, Denmark. ... that could prompt transition from cognitive normality to mild ... the Einstein Aging Study , established in 1980 ...
(Date:7/9/2014)... important organs in the human body. Its job is ... this is its synthesis function and that toxic ... its detoxification function. Lack of exercise and too much ... The resulting diseased cells can lead to inflammations, cancerous ... to the German Liver Foundation, over five million people ...
(Date:7/9/2014)... July 9, 2014 A study published in the ... Koninck and Robert Bonin, two researchers at Universit Laval, ... using a new method that involves rekindling pain so ... lead to novel means to alleviate chronic pain. , ... Laval and Institut universitaire en sant mentale de Qubec ...
(Date:7/8/2014)... Department of Defense,s Defense Advanced Research Projects Agency ... to $2.5 million to develop an implantable neural ... neurons within the brain to help restore memory, ... builds on the understanding that memory is a ... the brain encode information, store it and retrieve ...
Breaking Medicine News(10 mins):Health News:BU researchers relate arrests with HIV risk environment 2Health News:Investigators from Montefiore and Einstein to present data at 2014 Alzheimer's Association International Conference 2Health News:Investigators from Montefiore and Einstein to present data at 2014 Alzheimer's Association International Conference 3Health News:Telemedicine for patients with chronic liver diseases 2Health News:Telemedicine for patients with chronic liver diseases 3Health News:Discovery of a new means to erase pain 2Health News:DARPA selects Lawrence Livermore to develop world's first neural device to restore memory 2Health News:DARPA selects Lawrence Livermore to develop world's first neural device to restore memory 3
... Alliance Leverages the Adify Platform to Aggregate Leading ... Baby Boomers Caring for Aging Parents, SAN ... a comprehensive online resource for people caring,for aging ... ), the first vertical ad,network to reach baby ...
... 2008A team of Universit Laval researchers has shown that the ... brains of mice fed a diet rich in animal fat ... diets typical of most industrialized countries promote the development of ... of Aging . , To demonstrate this, the team led ...
... will add significant growth to the ... company,s first aid ... Bulletin Board: DSCI) announced today that it has secured two,additional ... the highly popular and growing segment of printed,bandages allows for ...
... Month, SANTA MONICA, Calif., Oct. 28 /PRNewswire/ - ... coined to express the benefits of keeping physically fit,but ... Numerous studies,have shown that regular brain training sessions -- ... help stave off dementia and even the,symptoms of Alzheimer,s ...
... study between leading researchers in academia and industry from US, Australian ... 293 cell growth and productivity using protein derived from colostrum. ... ... A recent breakthrough was published in the scientific journal In Vitro ...
... LeonardoMD is pleased to announce that Medical Reimbursement ... Web-based practice management software to automate their high-volume ... billing for several dozen providers using LeonardoMD over ... outcome. LeonardoMD takes pride in working with ...
Cached Medicine News:Health News:Caring.com Launches First Ad Network to Reach Family Caregivers 2Health News:A high-fat diet could promote the development of Alzheimer's 2Health News:Derma Sciences Announces 3-Year Licensing Deals for Highly Profitable Character Strip Bandages 2Health News:10 Surprising Facts About Using Brain Training to Fight Dementia 2Health News:10 Surprising Facts About Using Brain Training to Fight Dementia 3Health News:Global Scientific Research Collaboration Leads to Breakthrough in Culturing HEK 293 Cells in Animal-free Media 2Health News:Medical Reimbursement Consultants, Inc. Selects LeonardoMD for Web-based Medical Billing 2Health News:Medical Reimbursement Consultants, Inc. Selects LeonardoMD for Web-based Medical Billing 3
For use with cushioned neckband....
... PVC siliconized. Stainless steel spiral reinforced. X-ray ... use. Adjustable Flange: The flange can be ... the thickness of the cervical soft tissue. ... the flange is tightly connected to the ...
... The permanent flexible tube ... of the neck flange helps ... neck, chin, and stoma, enhancing ... ,Bivona® FlexTend™ tracheostomy tubes ...
Aire-Cuf® tracheostomy tubes are ideal for short-term to medium-term ventilator support....
Medicine Products: