Zobrazeno 1 - 10
of 19
pro vyhledávání: '"Monica Doria"'
Autor:
Rita Ferla, Fabio Dell’Aquila, Monica Doria, Maria Ferraiuolo, Alessia Noto, Fabiana Grazioli, Virginia Ammendola, Francesco Testa, Paolo Melillo, Carolina Iodice, Giulia Risca, Novella Tedesco, Pierre Romain le Brun, Enrico Maria Surace, Francesca Simonelli, Stefania Galimberti, Maria Grazia Valsecchi, Jean-Brice Marteau, Philippe Veron, Stefano Colloca, Alberto Auricchio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 396-411 (2023)
Gene therapy of Usher syndrome type 1B (USH1B) due to mutations in the large Myosin VIIA (MYO7A) gene is limited by the packaging capacity of adeno-associated viral (AAV) vectors. To overcome this, we have previously developed dual AAV8 vectors which
Externí odkaz:
https://doaj.org/article/61581d1038c94d21b99808688a92c247
Autor:
Xinhua Zhang, Mauro Giacca, Mauricio R. Alvira, Xavier León, Catherine Pythoud, Alberto Auricchio, Lorena Zentilin, Will Fountain, Marcus Müller, Christophe Darmon, Martin Lock, Olivier Bockstael, Hiroaki Mizukami, Eduard Ayuso, Keiya Ozawa, Brigitte Weins, Susan P. McGorray, Kai Gao, Barbara Leuchs, Monica Doria, Yvet Noordman, Fatima Bosch, J. Fraser Wright, K. Reed Clark, Catherine Melas, Philippe Moullier, Véronique Blouin, Stephanie Bucher, Liliane Tenenbaum, Juergen Dr Kleinschmidt, Marina Sumaroka, Richard O. Snyder, Abdelwahed Chtarto, Inge van der Linden, Guangping Gao, Richard T. Surosky
Publikováno v:
Human Gene Therapy
Human Gene Therapy, Mary Ann Liebert, 2014, 25 (11), pp.977-987. ⟨10.1089/hum.2014.057⟩
Human Gene Therapy, 2014, 25 (11), pp.977-987. ⟨10.1089/hum.2014.057⟩
Human Gene Therapy, vol. 25, no. 11, pp. 977-987
Human Gene Therapy, Mary Ann Liebert, 2014, 25 (11), pp.977-987. ⟨10.1089/hum.2014.057⟩
Human Gene Therapy, 2014, 25 (11), pp.977-987. ⟨10.1089/hum.2014.057⟩
Human Gene Therapy, vol. 25, no. 11, pp. 977-987
Gene therapy approaches using recombinant adeno-associated virus serotype 2 (rAAV2) and serotype 8 (rAAV8) have achieved significant clinical benefits. The generation of rAAV Reference Standard Materials (RSM) is key to providing points of reference
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f63fbfb64391b66e543280f2b0942e2c
https://europepmc.org/articles/PMC4236062/
https://europepmc.org/articles/PMC4236062/
Autor:
Edoardo Villani, Massimo Giunti, Roberto Giovannoni, Marialuisa Lavitrano, Alberto Auricchio, Francesco Viola, Enrico Maria Surace, U. Di Vicino, Settimio Rossi, Roberto Ratiglia, Elena Marrocco, Francesca Simonelli, Francesco Testa, Maria Laura Bacci, M. Della Corte, M. Crasta, Monica Doria, Claudio Mussolino, Simona Neglia
Publikováno v:
Gene Therapy
Gene Therapy; Vol 18
Gene Therapy; Vol 18
Recent success in clinical trials supports the use of adeno-associated viral (AAV) vectors for gene therapy of retinal diseases caused by defects in the retinal pigment epithelium (RPE). In contrast, evidence of the efficacy of AAV-mediated gene tran
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a089501c56937addf4cf7a303dcf424e
http://hdl.handle.net/11585/103209
http://hdl.handle.net/11585/103209
Autor:
Alberto Auricchio, Christel Rivière, Michael Korn, Karine Poulard, R. Jude Samulski, Thomas J. Conlon, Mahuya Bose, Steven M. Soltys, Richard O. Snyder, John A. Chiorini, Irina Korytov, Gabriele Lux, Monica Doria, Achille François, Sigrid D. Roosendaal, Christina Raupp, Bart van Montfort, Hiroaki Mizukami, Darby L. Thomas, Fatima Bosch, Tina Caton, J. Fraser Wright, E. Jeffrey Beecham, Abdelwahed Chtarto, Olga Zelenaia, Gabor Veres, Martin Lock, Véronique Blouin-Tavel, Christophe Darmon, Xavier León, Lorena Zentilin, K. Reed Clark, Mauro Giacca, Liliane Tenenbaum, Richard T. Surosky, Keiya Ozawa, Joyce D. Francis, Terence R. Flotte, Tina Philipsberg, Philippe Moullier, Barry J. Byrne, Eduard Ayuso, Catherine Melas, Barbara Leuchs, Susan P. McGorray, Marcus Müller, Anne Douar, Yili Xu
A recombinant adeno-associated virus serotype 2 Reference Standard Material (rAAV2 RSM) has been produced and characterized with the purpose of providing a reference standard for particle titer, vector genome titer, and infectious titer for AAV2 gene
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f9ac86e897d9378bb19926afec4680f4
https://hdl.handle.net/11368/2493555
https://hdl.handle.net/11368/2493555
Autor:
A Chand, M Hildinger, Alexander Bello, C Kranendonk, Alberto Auricchio, Kaylie Tran, Mariacarmela Allocca, D Beniac, Monica Doria, Gary P. Kobinger
Publikováno v:
Gene therapy. 16(11)
High antigenic compatibility and low toxicity is associated with xenograft transplantation of porcine tissues in immunodeficient human recipients. We hypothesized that adeno-associated viruses (AAVs) of porcine origin could be highly compatible to hu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b45ecc2d1609c4539772d81b4305aeb5
https://pubmed.ncbi.nlm.nih.gov/19626054
https://pubmed.ncbi.nlm.nih.gov/19626054
Autor:
Jean Bennett, Umberto Di Vicino, Alberto Auricchio, Pasqualina Colella, Marco Petrillo, Janet R. Sparrow, Luisa Cutillo, Maria Garcia-Hoyos, So Ra Kim, Tonia S. Rex, Monica Doria, Daniel Gibbs, David S. Williams, Albert M. Maguire, Mariacarmela Allocca
Publikováno v:
The Journal of clinical investigation. 118(5)
Vectors derived from adeno-associated virus (AAV) are promising for human gene therapy, including treatment for retinal blindness. One major limitation of AAVs as vectors is that AAV cargo capacity has been considered to be restricted to 4.7 kb. Here
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4511495a003b56a660a306ea2fea3dd3
https://pubmed.ncbi.nlm.nih.gov/18414684
https://pubmed.ncbi.nlm.nih.gov/18414684
Autor:
Anne M. Traas, Alberto Auricchio, Alessandra Tessitore, Karyn Cullen, Thomas O'Malley, Mark E. Haskins, Ping Wang, Monica Doria
Publikováno v:
Molecular Therapy. 11:S95
Mucopolysaccharidosis (MPS) VI is a lysosomal storage disease caused by deficient activity of N-acetylgalactosamine 4-sulfatase (4S) resulting in an inability to degrade the glycosaminoglycan (GAG) dermatan sulfate, which accumulates in the lysosomes
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d8356a8097f08b4e8f69b9a1598e7261
https://doi.org/10.1016/j.ymthe.2005.06.243
https://doi.org/10.1016/j.ymthe.2005.06.243
Autor:
Colella, P1, Trapani, I1, Cesi, G1, Sommella, A1, Manfredi, A1, Puppo, A1, Iodice, C1, Rossi, S2, Simonelli, F2, Giunti, M3, Bacci, M L3, Auricchio, A4
Publikováno v:
Gene Therapy. Apr2014, Vol. 21 Issue 4, p450-456. 7p.
Publikováno v:
Gene Therapy Weekly; 10/6/2023, p1253-1253, 1p
Autor:
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
Publikováno v:
Human Molecular Genetics; 12/1/2015, Vol. 24 Issue 23, p6811-6825, 15p