Forum for Science, Industry and Business
Sponsored by:     Siemens  n-tv 
Search our Site:

Topic (optional):

 

Home Reports Life Sciences Content

Standing up to paraplegia with gene therapy

next article
16.12.2005

 


Elena Rugarli and colleagues from the National Neurological Institute in Milan have used gene therapy to save sensory and skeletal muscle nerve fibers from degeneration in mice with hereditary spastic paraplegia (HSP). This strategy, reported online on December 15 in advance of print publication in the January 2006 issue of the Journal of Clinical Investigation, holds promise for many other disorders characterized by nerve degeneration due to loss of function of a known gene.


Hereditary spastic paraplegia (HSP), a neurodegenerative disorder caused by progressive loss of sensory and skeletal muscle nerve fibers (axons), is characterized by weakness, spasticity, and impaired function of the lower limbs. The disorder is often due to mutations in the gene encoding the paraplegin protein. HSP sufferers are ultimately confined to a wheelchair, and currently there is no cure for the disease. In the current study, Rugarli and colleagues have shown that a one-time delivery of normal paraplegin by a viral vector to the spinal motor neurons of mice with HSP, before the onset of symptoms, was able to save axons from degeneration for up to 10 months.

Delivery of this mitochondrial energy-dependent protease improved motor function in the mice and these data show that delivery of an intracellular protein to spinal motor neurons by gene transfer may be useful not only for the treatment of HSP patients but also for those individuals with other forms of peripheral nerve damage of known genetic origin.

TITLE: Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia

AUTHOR CONTACT:
Elena I. Rugarli
National Neurological Institute, Milan, Italy
Phone: 39-02-23942614, Fax: 39-02-23942619, E-mail: rugarli@istituto-besta.it

Brooke Grindlinger | Source: EurekAlert!
Further information: www.the-jci.org
www.the-jci.org/article.php?id=26210

next article

More articles from Life Sciences:

nachricht First reconstitution of an epidermis from human embryonic stem cells
23.11.2009 | INSERM (Institut national de la santé et de la recherche médicale)

nachricht Causative gene of a rare disorder discovered by sequencing only protein-coding regions of genome
23.11.2009 | University of Washington

All articles from Life Sciences >>>

B2B Search

Product / Service
Company / Organisation

Latest News

UCSB physicists move 1 step closer to quantum computing

23.11.2009 | Physics and Astronomy

Fat around the middle increases the risk of dementia

23.11.2009 | Studies and Analyses

New discovery about the formation of new brain cells

23.11.2009 | Health and Medicine

VideoLinks

Event News

Multidisciplinary meeting on Urological Cancers aims to benefit cancer patients

20.11.2009 | Event News

'Golden Age' for clinical psychology in Northern Ireland

20.11.2009 | Event News

New Perspectives in Marine Anti-Fouling Research

11.11.2009 | Event News