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:6/9/2016)... an innovation leader in attendance control systems is proud to announce the introduction of ... make sure the right employees are actually signing in, and to even control the ... ... ... ...
(Date:6/3/2016)... 2016 Das DOTM ... Nepal hat ein 44 Millionen ... Kennzeichen, einschließlich Personalisierung, Registrierung und IT-Infrastruktur, an ... und Implementierung von Identitätsmanagementlösungen. Zahlreiche renommierte internationale ... teilgenommen, aber Decatur wurde als konformste und ...
(Date:6/2/2016)... June 2, 2016 Perimeter Surveillance ... Unmanned Systems, Physical Infrastructure, Support & Other Service  ... visiongain offers comprehensive analysis of the global ... will generate revenues of $17.98 billion in 2016. ... Inc, a leader in software and hardware technologies for ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... NC (PRWEB) , ... June 27, 2016 , ... ... just published their findings on what they believe could be a new and ... of the new research. Click here to read it now. , ...
(Date:6/27/2016)... ... June 27, 2016 , ... Newly created ... and solutions to the healthcare market. The company's primary focus is on new ... and marketing strategies that are necessary to help companies efficiently bring their products ...
(Date:6/27/2016)... PHILADELPHIA , June 27, 2016  Liquid ... today announced the funding of a Sponsored Research ... study circulating tumor cells (CTCs) from cancer patients.  ... changes in CTC levels correlate with clinical outcomes ... therapies. These data will then be employed to ...
(Date:6/24/2016)... TOKYO , June 24, 2016  Regular discussions on ... to take place between the two entities said Poloz. ... in Ottawa , he pointed to the ... and the federal government. ... Poloz said, "Both institutions have common economic goals, why not ...
Breaking Biology Technology: