Sfoglia per Rivista GENE THERAPY
Absence of an intrathecal immune reaction to a helper-dependent adenoviral vector delivered into the cerebrospinal fluid of non-human primates.
2008 Butti, E; Bergami, A; Recchia, Alessandra; Brambilla, E; Franciotta, D; Cattalini, A; Stornaiuolo, A; Lachapelle, F; Comi, G; Mavilio, Fulvio; Martino, G; Furlan, R.
Corrective gene transfer of keratinocytes from patients with junctional epidermolysis bullosa restores assembly of hemidesmosomes in reconstructed epithelia
1998 Vailly, J; Gagnoux Palacios, L; Dell'Ambra, E; Roméro, C; Pinola, M; Zambruno, G; De Luca, Michele; Ortonne, J. P; Meneguzzi, G.
CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting
2016 Courtney, D. G.; Moore, J. E.; Atkinson, S. D.; Maurizi, E.; Allen, E. H. A.; Pedrioli, D. M. L.; Mclean, W. H. I.; Nesbit, M. A.; Moore, C. B. T.
Developing gene and cell therapies for rare diseases: An opportunity for synergy between academia and industry
2017 Mavilio, F.
Efficient in vitro and in vivo gene regulation of a retrovirally delivered pro-apoptotic factor under the control of the Drosophila HSP70 promoter
2001 Romano, G; Reiss, K; Tu, X; Peruzzi, F; Belletti, B; Wang, J. Y.; Zanocco Marani, Tommaso; Baserga, R.
IL4 gene delivery to the CNS recruits regulatory T cells and induces clinical recovery in mouse models of multiple sclerosis
2008 Butti, E; Bergami, A; Recchia, Alessandra; Brambilla, E; Del Carro, U; Amadio, S; Cattalini, A; Esposito, M; Stornaiuolo, A; Comi, G; Pluchino, S; Mavilio, Fulvio; Martino, G; Furlan, R.
Metabolically stabilized long-circulating PEGylated polyacridine peptide polyplexes mediate hydrodynamically stimulated gene expression in liver
2011 Fernandez, C. A.; Baumhover, N. J.; Duskey, J. T.; Khargharia, S.; Kizzire, K.; Ericson, M. D.; Rice, K. G.
Risk assessment in skin gene therapy: Viral-cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors
2011 Almarza, D.; Bussadori, G.; Navarro, M.; Mavilio, F.; Larcher, F.; Murillas, R.
Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes
2013 Cavazza, A.; Cocchiarella, F.; Bartholomae, C.; Schmidt, M.; Pincelli, C.; Larcher, F.; Mavilio, F.
The importance of epidermal stem cells in keratinocyte-mediated gene therapy
1997 De Luca, Michele; Pellegrini, Graziella
The uptake mechanism of PEGylated DNA polyplexes by the liver influences gene expression
2014 Khargharia, S.; Baumhover, N. J.; Crowley, S. T.; Duskey, J.; Rice, K. G.
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