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Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.
Visigalli, Ilaria; Delai, Stefania; Ferro, Francesca; Cecere, Francesca; Vezzoli, Michela; Sanvito, Francesca; Chanut, Franck; Benedicenti, Fabrizio; Spinozzi, Giulio; Wynn, Rob; Calabria, Andrea; Naldini, Luigi; Montini, Eugenio; Cristofori, Patrizia; Biffi, Alessandra.
Afiliação
  • Visigalli I; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Delai S; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Ferro F; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Cecere F; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Vezzoli M; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Sanvito F; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Chanut F; 2 Department of Pathology, IRCCS San Raffaele Scientific Institute , Milan, Italy.
  • Benedicenti F; 3 Glaxo Smith Kline R&D UK Safety Assessment , Stevenage, United Kingdom.
  • Spinozzi G; 5 Sanofi , Montpellier, France.
  • Wynn R; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Calabria A; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Naldini L; 6 Royal Manchester Children's Hospital , Manchester, United Kingdom .
  • Montini E; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Cristofori P; 1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) , Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy.
  • Biffi A; 4 Vita Salute San Raffaele University , Milan, Italy.
Hum Gene Ther ; 27(10): 813-829, 2016 10.
Article em En | MEDLINE | ID: mdl-27431943
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Mucopolissacaridose I / Transplante de Células-Tronco Hematopoéticas / Iduronidase Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Hum Gene Ther Assunto da revista: GENETICA MEDICA / TERAPEUTICA Ano de publicação: 2016 Tipo de documento: Article País de afiliação: Itália
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Mucopolissacaridose I / Transplante de Células-Tronco Hematopoéticas / Iduronidase Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Hum Gene Ther Assunto da revista: GENETICA MEDICA / TERAPEUTICA Ano de publicação: 2016 Tipo de documento: Article País de afiliação: Itália