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Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin A under control of the colony-stimulating factor-1 receptor promoter

Galactosialidosis (GS) is a human neurodegenerative disease caused by a deficiency of lysosomal protective protein/cathepsin A (PPCA). The GS mouse model resembles the severe human condition, resulting in nephropathy, ataxia, and premature death. To rescue the disease phenotype, GS mice were transpl...

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Détails bibliographiques
Auteurs principaux: Hahn, Christopher N., del Pilar Martin, Maria, Zhou, Xiao-Yan, Mann, Linda W., d’Azzo, Alessandra
Format: Artigo
Langue:Inglês
Publié: The National Academy of Sciences 1998
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Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC24544/
https://ncbi.nlm.nih.gov/pubmed/9843984
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