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Skeletal Muscle|September 9, 2015
Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1ATeuta Domi, Emanuela Porrello, Daniele Velardo, et al.
Pediatric Hematology and Oncology|July 8, 2024
Oncological pediatric early warning score: a dedicated tool to predict patient's clinical deterioration and need for pediatric intensive care treatmentTeresa Maccarana, Marta Pillon, Veronica Bertozzi, et al.
Neurobiology of Disease|March 26, 2009
Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195Ilaria Visigalli, Rosa Maria Moresco, Sara Belloli, et al.
Annals of Neurology|November 19, 2013
Metallothioneins as dynamic markers for brain disease in lysosomal disordersMartina Cesani, Eleonora Cavalca, Romina Macco, et al.
The Journal of Clinical Investigation|November 3, 2006
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in miceAlessandra Biffi, Alessia Capotondo, Stefania Fasano, et al.
Science Translational Medicine|November 19, 2010
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophyBernhard Gentner, Ilaria Visigalli, Hidefumi Hiramatsu, et al.
Nanomedicine (London, England)|May 15, 2014
Cellular magnetic resonance with iron oxide nanoparticles: long-term persistence of SPIO signal in the CNS after transplanted cell deathChiara Cianciaruso, Antonella Pagani, Cristina Martelli, et al.
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