Standing up to paraplegia with gene therapyDecember 16, 2005Elena 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. Journal of Clinical Investigation |
|||||||||||||||||||||
| Related Paraplegia Current Events and Paraplegia News Articles Little-known protein found to be key player Italian and U.S. biologists this week report that a little-understood protein previously implicated in a rare genetic disorder plays an unexpected and critical role in building and maintaining healthy cells. Medical study shows epidurals and spinal anesthetics are safer than previously reported The largest ever prospective study into the major complications of epidurals and spinal anaesthetics published in the British Journal of Anaesthesia today (Monday 12 January 2009) concludes that previous studies have over-estimated the risks of severe complications of these procedures. Motor neuron disease and toxic substances: Possible link? Motor neuron disease is a rare, devastating illness in which nerve cells that carry brain signals to muscles gradually deteriorate. One form of it, Lou Gehrig's disease or ALS (amyotrophic lateral sclerosis), is familiar to the public in the lives of scientist Stephen Hawking and Morrie Schwartz, about whom Mitch Albom's "Tuesdays with Morrie" was written. Marathon mice elucidate little-known muscle type Researchers report in the January issue of the journal Cell Metabolism, published by Cell Press, the discovery of a genetic "switch" that drives the formation of a poorly understood type of muscle. Moreover, they found, animals whose muscles were full of the so-called IIX fibers were able to run farther and at higher work loads than normal mice could. More effective reporting needed on spinal manipulation in children Few serious harmful events stemming from spinal manipulation in children have been reported compared to the number of manipulations delivered. Neurons find their place in the developing nervous system with the help of a sticky molecule The brain, that exquisite network of billions of communicating cells, starts to take form with the genesis of nerve cells. Most newborn nerve cells, also called neurons, must travel from their birthplace to the position they will occupy in the adult brain. Wheelchair technology & spinal cord injury The current issue of the Journal of Rehabilitation Research and Development (JRRD) is dedicated to spinal cord injury and wheelchair technology. Nerve cells' power plants caught in a traffic jam Nerve cells need lots of energy to work properly, and the energy needs to be delivered to the right place at the right time. By inducing a mutation in fruit flies, researchers have figured out that a particular gene governs the movement of cells' energy-producing units, called mitochondria. Profile of the Aging Kidney: PLoS Biology Press Release A Global View of Gene Expression in the Aging Kidney Hopes of a New Treatment Approach for Paralysis Scientists at the Deutsches Krebsforschungszentrum (German Cancer Research Center, Heidelberg) were recently able to show in experiments with mice that a paralysis can be reversed by blocking programmed cell death. Paralysis in the form of paraplegia and quadriplegia is usually the result of traumatic spinal cord injuries, but can also be caused by tumors. When the spinal cord is cut off, information sent by the brain can no longer be transmitted to the limbs. Biological processes such as programmed cell death, or apoptosis, take place after the actual moment of injury of the nerve cords and lead to further destruction of the damaged tissue and thus to permanent paralysis. More Paraplegia Current Events and Paraplegia News Articles |
|||||||||||||||||||||
|
|||||||||||||||||||||
|
|||||||||||||||||||||