An engineered U1 small nuclear RNA rescues splicing‐defective coagulation F7 gene expression in mice
Balestra, D., Faella, A., Margaritis, P., Cavallari, N., Pagani, F., Bernardi, F., Arruda, V. R., Pinotti, M.
Published in Journal of thrombosis and haemostasis (01.02.2014)
Published in Journal of thrombosis and haemostasis (01.02.2014)
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An engineered U1 small nuclear RNA rescues splicing defective coagulation F7 gene expression in mice
Balestra, D, Faella, A, Margaritis, P, Cavallari, N, Pagani, F, Bernardi, F, Arruda, V R, Pinotti, M
Published in Journal of thrombosis and haemostasis (01.02.2014)
Published in Journal of thrombosis and haemostasis (01.02.2014)
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Overcoming preexisting humoral immunity to AAV using capsid decoys
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Published in Science translational medicine (17.07.2013)
Published in Science translational medicine (17.07.2013)
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Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses
Cotugno, Gabriella, Tessitore, Alessandra, Capalbo, Anita, Annunziata, Patrizia, Strisciuglio, Caterina, Faella, Armida, Aurilio, Michela, Di Tommaso, Maurizio, Russo, Fabio, Mancini, Antonio, De Leonibus, Elvira, Aloj, Luigi, Auricchio, Alberto
Published in Human gene therapy (01.05.2010)
Published in Human gene therapy (01.05.2010)
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