Navigation Links
Gene therapy slows progression of fatal neurodegenerative disease in children
Date:5/13/2008

New Rochelle, NY, May 13, 2008Gene 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 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.


'/>"/>

Contact: Vicki Cohn
vcohn@liebertpub.com
914-740-2156
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert

Related biology news :

1. UC Davis stem researchers demonstrate safety of gene therapy using adult stem cells
2. Cell-based therapy shows promise in patients with Parkinsons disease
3. Cancer immunotherapy shows long-term promise in lung cancer
4. Effective cancer immune therapy through order in the blood vessels
5. New chemotherapy combo produces side effects, but no extra efficacy, in early breast cancer patients
6. Biomarkers allow doctors to match therapy to patient
7. MU researcher links hormone replacement therapy to breast cancer
8. Potential new target for multiple sclerosis therapy
9. Umbilical cord blood cell therapy may reduce signs and symptoms of Alzheimers disease
10. Umbilical cord blood cell therapy in an animal model of Alzheimers disease
11. Tumor-targeting viral therapy slows neuroblastoma, malignant peripheral nerve sheath tumors
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/18/2017)... -- Socionext Inc., a global expert in SoC-based imaging and computing solutions, ... which features the company,s hybrid codec technology. A demonstration utilizing TeraFaces ... will be showcased during the upcoming Medtec Japan at Tokyo Big ... Las Vegas Convention Center April 24-27. ... Click here for an image of ...
(Date:4/11/2017)... Apr. 11, 2017 Research and Markets has ... report to their offering. ... The global eye tracking market to grow at a CAGR of ... Eye Tracking Market 2017-2021, has been prepared based on an in-depth ... market landscape and its growth prospects over the coming years. The ...
(Date:4/5/2017)... YORK , April 5, 2017 Today ... is announcing that the server component of the HYPR ... known for providing the end-to-end security architecture that empowers ... HYPR has already secured over 15 million ... makers including manufacturers of connected home product suites and ...
Breaking Biology News(10 mins):
(Date:10/10/2017)... ... October 10, 2017 , ... ... targeted antibody-drug conjugate (ADC) therapeutics, today confirmed licensing rights that give it ... Nanoparticle), a technology developed in collaboration with Children’s Hospital Los Angeles (CHLA). ...
(Date:10/10/2017)... ... October 10, 2017 , ... ... process optimization firm for the life sciences and healthcare industries, announces a presentation ... San Francisco. , The presentation, “Automating GxP Validation for Agile Cloud Platforms,” will ...
(Date:10/9/2017)... ... October 09, 2017 , ... The Giving Tree ... products targeting the needs of consumers who are incorporating medical marijuana into their ... Phoenix, Arizona. , As operators of two successful Valley dispensaries, The Giving Tree’s ...
(Date:10/6/2017)... ... October 06, 2017 , ... The HealthTech Venture ... sector at their fourth annual Conference where founders, investors, innovative practitioners and collaborators ... ELEVATE pitch competition showcasing early stage digital health and med tech companies. , ...
Breaking Biology Technology: