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One or more keywords matched the following items that are connected to Biffi, Alessandra
Item TypeName
Academic Article Human hematopoietic stem cells in gene therapy: pre-clinical and clinical issues.
Academic Article Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.
Academic Article Novel candidate disease for gene therapy: metachromatic leukodystrophy.
Academic Article Gene therapy of storage disorders by retroviral and lentiviral vectors.
Academic Article Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
Academic Article Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.
Academic Article Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.
Academic Article Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Academic Article Gene therapy for leukodystrophies.
Academic Article Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy.
Academic Article Gene therapy for lysosomal storage disorders: a good start.
Academic Article Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Academic Article Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.
Academic Article Gene Therapy as a Curative Option for ß-Thalassemia.
Academic Article Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.
Academic Article Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings.
Academic Article Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease.
Academic Article Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
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  • Gene Therapy
Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.