Engraftable human neural stem cells

US6680198B1 · kind B1 · utility

Assignees

• Children's Medical Center Corporation · US
• University of British Columbia · CA
• The Trustees of the University of Pennsylvania · US

Inventors

• Evan Snyder · Village of La Jolla, US
• John H. Wolfe · Blue Bell, US
• Seung U. Kim · Vancouver, CA

Key dates

• Filing date: Sep 20, 1999
• Grant date: Jan 20, 2004
• Expiry date: Sep 20, 2019

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2510/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Stable clones of neural stem cells (NSCs) have been isolated from the human fetal telencephalon. In vitro, these self-renewing clones (affirmed by retroviral insertion site) can spontaneously give rise to all 3 fundamental neural cell types (neurons, oligodendrocytes, astrocytes). Following transplantation into germinal zones of the developing newborn mouse brain, they, like their rodent counterparts, can participate in aspects of normal development, including migration along well-established migratory pathways to disseminated CNS regions, differentiation into multiple developmentally- and regionally-appropriate cell types in response to microenvironmental cues, and non-disruptive, non-tumorigenic interspersion with host progenitors and their progeny. Readily genetically engineered prior to transplantation, human NSCs are capable of expressing foreign transgenes in vivo in these disseminated locations. Further supporting their potential for gene therapeutic applications, the secretory products from these NSCs can cross-correct a prototypical genetic metabolic defect in abnormal neurons and glia in vitro as effectively as do murine NSCs. Finally, human cells appear capable of replac…