Sfoglia per Autore
Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease
2016 Alliegro, Marialuisa; Ferla, Rita; Nusco, Edoardo; DE LEONIBUS, Chiara; Settembre, Carmine; Auricchio, Alberto
Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease
2015 Trapani, Ivana; Toriello, Elisabetta; de Simone, Sonia; Colella, Pasqualina; Iodice, Carolina; Polishchuk, Elena V; Sommella, Andrea; Colecchi, Linda; Rossi, Settimio; Simonelli, Francesca; Giunti, Massimo; Bacci, Maria L; Polishchuk, Roman S; Auricchio, Alberto
Methods and compositions for treating genetic eye diseases
2015 Davidson, B; Chen, Yh; Auricchio, A
Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.
2015 Ferla, R; Claudiani, P; Savarese, M; Kozarsky, K.; Parini, R; Scarp, a M; Donati, Ma; Sorge, G; Hopwood, Jj; Parenti, Giancarlo; Fecarotta, S; Nigro, V; Sivri, Hs; Van Der Ploeg, A; Andria, Generoso; BRUNETTI PIERRI, Nicola; Auricchio, Alberto
Gene Therapy of ABCA4-Associated Diseases.
2015 Auricchio, Alberto; Trapani, I; Allikmets, R.
Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression.
2015 Biferi, Mg; Nicoletti, C; Falcone, G; Puggioni, Em; Passaro, N; Mazzola, A; Pajalunga, D; Zaccagnini, G; Rizzuto, E; Auricchio, Alberto; Zentilin, L; De Luca, G; Giacca, M; Martelli, F; Musio, A; Musarò, A; Crescenzi, M.
SEPN1, an endoplasmic reticulum-localized selenoprotein linked to skeletal muscle pathology, counteracts hyperoxidation by means of redox-regulating SERCA2 pump activity.
2015 Marino, M; Stoilova, T; Giorgi, C; Bachi, A; Cattaneo, A; Auricchio, Alberto; Pinton, P; Zito, E.
Advancing therapeutic strategies for inherited retinal degeneration: Recommendations from the monaciano symposium
2015 Thompson, D. A.; Ali, R. R.; Banin, E.; Branham, K. E.; Flannery, J. G.; Gamm, D. M.; Hauswirth, W. W.; Heckenlively, J. R.; Iannaccone, A.; Thiran Jayasundera, K.; Khan, N. W.; Molday, R. S.; Pennesi, M. E.; Reh, T. A.; Weleber, R. G.; Zacks, D. N.; Auricchio, A.
Gene therapy of inherited retinal degenerations: prospects and challenges.
2015 Trapani, I; Banfi, S; Simonelli, F; Surace, Enrico Maria; Auricchio, Alberto
Cross-talk between androgen receptor/filamin A and TrkA regulates neurite outgrowth in PC12 cells
2015 Di Donato, Marzia; Bilancio, Antonio; D'Amato, Loredana; Claudiani, Pamela; Oliviero, Maria Antoniett; Barone, Maria Vittoria; Auricchio, Alberto; Appella, Ettore; Migliaccio, Antimo; Auricchio, Ferdinando; Castoria, Gabriella
Multiple vector system and uses thereof.
2015 Colella, P; Auricchio, A; Trapani, I
Genetic Analysis Reveals a Longevity-Associated Protein Modulating Endothelial Function and Angiogenesis.
2015 Villa, F; Carrizzo, A; Spinelli, Cc; Ferrario, A; Malovini, A; Maciag, A; Damato, A; Auricchio, Alberto; Spinetti, G; Sangalli, E; Dang, Z; Madonna, M; Ambrosio, M; Sitia, L; Bigini, P; Calì, G; Schreiber, S; Perls, T; Fucile, S; Mulas, F; Nebel, A; Bellazzi, R; Madeddu, Pr; Vecchione, C; Puca, Aa
Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease.
2014 Ferla, R; Claudiani, P; Cotugno, G; Saccone, P; De Leonibus, E; Auricchio, Alberto
Novel adeno-associated viruses derived from pig tissues transduce most major organs in mice.
2014 Bello, A; Chand, A; Aviles, J; Soule, G; Auricchio, Alberto; Kobinger, Gp
Vector platforms for gene therapy of inherited retinopathies.
2014 Trapani, I; Puppo, A; Auricchio, Alberto
Gene Therapy Using a Liver-targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE.
2014 Torres Torronteras, J; Viscomi, C; Cabrera P?rez, R; C?mara, Y; Di Meo, I; Barquinero, J; Auricchio, Alberto; Pizzorno, G; Hirano, M; Zeviani, M; Mart?, R.
Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI).
2014 Saccone, P; Cotugno, G; Russo, F; Mastrogiacomo, R; Tessitore, A; Auricchio, Alberto; De Leonibus, E.
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.
2014 Ayuso, E; Blouin, V; Lock, M; Mcgorray, S; Leon, X; Alvira, Mr; Auricchio, Alberto; Bucher, S; Chtarto, A; Clark, Kr; Darmon, C; Doria, M; Fountain, W; Gao, G; Gao, K; Giacca, M; Kleinschmidt, J; Leuchs, B; Melas, C; Mizukami, H; Müller, M; Noordman, Y; Bockstael, O; Ozawa, K; Pythoud, C; Sumaroka, M; Surosky, R; Tenenbaum, L; van der Linden, I; Weins, B; Wright, Jf; Zhang, X; Zentilin, L; Bosch, F; Snyder, Ro; Moullier, P.
AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure.
2014 Bottani, E; Giordano, C; Civiletto, G; Di Meo, I; Auricchio, Alberto; Ciusani, E; Marchet, S; Lamperti, C; D'Amati, G; Viscomi, C; Zeviani, M.
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.
2014 Colella, P; Trapani, I; Cesi, G; Sommella, A; Manfredi, A; Puppo, A; Iodice, C; Rossi, S; Simonelli, F; Giunti, M; Bacci, Ml; Auricchio, Alberto
| Titolo | Tipologia | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|---|
| Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease | 1.1 Articolo in rivista | 2016 | Alliegro, Marialuisa; Ferla, Rita; Nusco, Edoardo; DE LEONIBUS, Chiara; Settembre, Carmine; Auricchio, Alberto | |
| Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease | 1.1 Articolo in rivista | 2015 | Trapani, Ivana; Toriello, Elisabetta; de Simone, Sonia; Colella, Pasqualina; Iodice, Carolina; Polishchuk, Elena V; Sommella, Andrea; Colecchi, Linda; Rossi, Settimio; Simonelli, Francesca; Giunti, Massimo; Bacci, Maria L; Polishchuk, Roman S; Auricchio, Alberto | |
| Methods and compositions for treating genetic eye diseases | 6.1 Brevetto | 2015 | Davidson, B; Chen, Yh; Auricchio, A | |
| Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial. | 1.1 Articolo in rivista | 2015 | Ferla, R; Claudiani, P; Savarese, M; Kozarsky, K.; Parini, R; Scarp, a M; Donati, Ma; Sorge, G; Hopwood, Jj; Parenti, Giancarlo; Fecarotta, S; Nigro, V; Sivri, Hs; Van Der Ploeg, A; Andria, Generoso; BRUNETTI PIERRI, Nicola; Auricchio, Alberto | |
| Gene Therapy of ABCA4-Associated Diseases. | 1.1 Articolo in rivista | 2015 | Auricchio, Alberto; Trapani, I; Allikmets, R. | |
| Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression. | 1.1 Articolo in rivista | 2015 | Biferi, Mg; Nicoletti, C; Falcone, G; Puggioni, Em; Passaro, N; Mazzola, A; Pajalunga, D; Zaccagnini, G; Rizzuto, E; Auricchio, Alberto; Zentilin, L; De Luca, G; Giacca, M; Martelli, F; Musio, A; Musarò, A; Crescenzi, M. | |
| SEPN1, an endoplasmic reticulum-localized selenoprotein linked to skeletal muscle pathology, counteracts hyperoxidation by means of redox-regulating SERCA2 pump activity. | 1.1 Articolo in rivista | 2015 | Marino, M; Stoilova, T; Giorgi, C; Bachi, A; Cattaneo, A; Auricchio, Alberto; Pinton, P; Zito, E. | |
| Advancing therapeutic strategies for inherited retinal degeneration: Recommendations from the monaciano symposium | 1.1 Articolo in rivista | 2015 | Thompson, D. A.; Ali, R. R.; Banin, E.; Branham, K. E.; Flannery, J. G.; Gamm, D. M.; Hauswirth, W. W.; Heckenlively, J. R.; Iannaccone, A.; Thiran Jayasundera, K.; Khan, N. W.; Molday, R. S.; Pennesi, M. E.; Reh, T. A.; Weleber, R. G.; Zacks, D. N.; Auricchio, A. | |
| Gene therapy of inherited retinal degenerations: prospects and challenges. | 1.1 Articolo in rivista | 2015 | Trapani, I; Banfi, S; Simonelli, F; Surace, Enrico Maria; Auricchio, Alberto | |
| Cross-talk between androgen receptor/filamin A and TrkA regulates neurite outgrowth in PC12 cells | 1.1 Articolo in rivista | 2015 | Di Donato, Marzia; Bilancio, Antonio; D'Amato, Loredana; Claudiani, Pamela; Oliviero, Maria Antoniett; Barone, Maria Vittoria; Auricchio, Alberto; Appella, Ettore; Migliaccio, Antimo; Auricchio, Ferdinando; Castoria, Gabriella | |
| Multiple vector system and uses thereof. | 6.1 Brevetto | 2015 | Colella, P; Auricchio, A; Trapani, I | |
| Genetic Analysis Reveals a Longevity-Associated Protein Modulating Endothelial Function and Angiogenesis. | 1.1 Articolo in rivista | 2015 | Villa, F; Carrizzo, A; Spinelli, Cc; Ferrario, A; Malovini, A; Maciag, A; Damato, A; Auricchio, Alberto; Spinetti, G; Sangalli, E; Dang, Z; Madonna, M; Ambrosio, M; Sitia, L; Bigini, P; Calì, G; Schreiber, S; Perls, T; Fucile, S; Mulas, F; Nebel, A; Bellazzi, R; Madeddu, Pr; Vecchione, C; Puca, Aa | |
| Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease. | 1.1 Articolo in rivista | 2014 | Ferla, R; Claudiani, P; Cotugno, G; Saccone, P; De Leonibus, E; Auricchio, Alberto | |
| Novel adeno-associated viruses derived from pig tissues transduce most major organs in mice. | 1.1 Articolo in rivista | 2014 | Bello, A; Chand, A; Aviles, J; Soule, G; Auricchio, Alberto; Kobinger, Gp | |
| Vector platforms for gene therapy of inherited retinopathies. | 1.1 Articolo in rivista | 2014 | Trapani, I; Puppo, A; Auricchio, Alberto | |
| Gene Therapy Using a Liver-targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE. | 1.1 Articolo in rivista | 2014 | Torres Torronteras, J; Viscomi, C; Cabrera P?rez, R; C?mara, Y; Di Meo, I; Barquinero, J; Auricchio, Alberto; Pizzorno, G; Hirano, M; Zeviani, M; Mart?, R. | |
| Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI). | 1.1 Articolo in rivista | 2014 | Saccone, P; Cotugno, G; Russo, F; Mastrogiacomo, R; Tessitore, A; Auricchio, Alberto; De Leonibus, E. | |
| Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. | 1.1 Articolo in rivista | 2014 | Ayuso, E; Blouin, V; Lock, M; Mcgorray, S; Leon, X; Alvira, Mr; Auricchio, Alberto; Bucher, S; Chtarto, A; Clark, Kr; Darmon, C; Doria, M; Fountain, W; Gao, G; Gao, K; Giacca, M; Kleinschmidt, J; Leuchs, B; Melas, C; Mizukami, H; Müller, M; Noordman, Y; Bockstael, O; Ozawa, K; Pythoud, C; Sumaroka, M; Surosky, R; Tenenbaum, L; van der Linden, I; Weins, B; Wright, Jf; Zhang, X; Zentilin, L; Bosch, F; Snyder, Ro; Moullier, P. | |
| AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure. | 1.1 Articolo in rivista | 2014 | Bottani, E; Giordano, C; Civiletto, G; Di Meo, I; Auricchio, Alberto; Ciusani, E; Marchet, S; Lamperti, C; D'Amati, G; Viscomi, C; Zeviani, M. | |
| Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors. | 1.1 Articolo in rivista | 2014 | Colella, P; Trapani, I; Cesi, G; Sommella, A; Manfredi, A; Puppo, A; Iodice, C; Rossi, S; Simonelli, F; Giunti, M; Bacci, Ml; Auricchio, Alberto |
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