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 toward the development of treatments for this group of underserved inherited neurodegenerative disorders," says James M. Wilson, MD, PhD, Editor-in-Chief and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, at the University of Pennsylvania School of Medicine, in Philadelphia.

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and the British Society for Gene Therapy, is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of contents and a free sample issue may be viewed online.

Mary Ann Liebert, Inc. (http://www.liebertpub.com), is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cloning and Stem Cells. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 60 journals, books, and newsmagazines is available online.

Contact: Vicki Cohn, Mary Ann Liebert, Inc., (914) 740-2100, ext. 2156, vcohn@liebertpub.com


'/>"/>
SOURCE Mary Ann Liebert, Inc.
Copyright©2008 PR Newswire.
All rights reserved

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:9/27/2017)... NEW YORK , Sept. 27, 2017  DarioHealth Corp. ... big data solutions, today announced that its MyDario product is expected to ... TV listings for when The Dr. Oz Show airs in your area: ... The nine-time Emmy award-winning, ... ...
(Date:9/25/2017)... , Sept. 25, 2017  EpiVax, Inc., a ... design, and immune-engineering today announced the launch of ... development of personalized therapeutic cancer vaccines. EpiVax has ... exclusive access to enabling technologies to the new ... will lead EpiVax Oncology as Chief Executive Officer. ...
(Date:9/22/2017)... , Sept. 22, 2017  As the latest Obamacare ... Bill Cassidy (R-LA) and Lindsey Graham ... that the medical device industry is in an odd ... tax, the 2.3% excise tax on medical device sales ... also want covered patients, increased visits and hospital customers ...
Breaking Medicine Technology:
(Date:10/13/2017)... Pekin, IL (PRWEB) , ... October 13, 2017 ... ... Foundation, which established the certification process to promote standards of excellence for the ... iaedp™ Symposium, scheduled for March 22 – 25, 2018 in Orlando, Florida at ...
(Date:10/13/2017)... ... , ... Many families have long-term insurance that covers care for a family ... for care if the client has a cognitive impairment diagnosis. , “What this ... often waived, so the benefits from their insurance start immediately,” said Mechell Vieira, owner ...
(Date:10/13/2017)... ... 2017 , ... Talented host, actor Rob Lowe, is introducing ... new episode of "Success Files," which is an award-winning educational program broadcasted on ... subject in-depth with passion and integrity. , Sciatica occurs when the sciatic nerve ...
(Date:10/12/2017)... ... October 12, 2017 , ... ... sleep apnea using cutting-edge Oventus O2Vent technology. As many as 18 ... characterized by frequent cessation in breathing. Oral appliances can offer significant relief to ...
(Date:10/12/2017)... ... October 12, 2017 , ... ... leader in post-acute health care, have expanded their existing home health joint venture ... , AccentCare has been operating a joint venture home health company with Asante, ...
Breaking Medicine News(10 mins):