alexa Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain.
Physicaltherapy & Rehabilitation

Physicaltherapy & Rehabilitation

International Journal of Physical Medicine & Rehabilitation

Author(s): Snyder EY, Taylor RM, Wolfe JH

Abstract Share this page

Abstract Many metabolic diseases affecting the central nervous system are refractory to treatment because the blood-brain barrier restricts entry of therapeutic molecules. It may be possible to deliver therapeutic gene products directly to the brain by transplantation of neural progenitor cells, which can integrate into the murine central nervous system in a cytoarchitecturally appropriate manner. We tested this approach in mucopolysaccharidosis VII (Sly disease), a lysosomal storage disorder of humans, dogs and mice caused by an inherited deficiency of beta-glucuronidase. Lysosomal accumulation of glycosaminoglycans occurs in the brain and other tissues, causing a fatal progressive degenerative disorder, including mental retardation. Treatments are designed to provide a source of normal enzyme for uptake by diseased cells. We report here that by transplanting beta-glucuronidase-expressing neural progenitors into the cerebral ventricles of newborn mice, donor cells engrafted throughout the neuraxis. At maturity, donor-derived cells were present as normal constituents of diverse brain regions. beta-Glucuronidase activity was expressed along the entire neuraxis, resulting in widespread correction of lysosomal storage in neurons and glia in affected mice. This article was published in Nature and referenced in International Journal of Physical Medicine & Rehabilitation

Relevant Expert PPTs

Relevant Speaker PPTs

Recommended Conferences

Relevant Topics

Peer Reviewed Journals
 
Make the best use of Scientific Research and information from our 700 + peer reviewed, Open Access Journals
International Conferences 2017-18
 
Meet Inspiring Speakers and Experts at our 3000+ Global Annual Meetings

Contact Us

 
© 2008-2017 OMICS International - Open Access Publisher. Best viewed in Mozilla Firefox | Google Chrome | Above IE 7.0 version
adwords