Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

Autor: Maria Rosa Lidonnici, Ylenia Paleari, Francesca Tiboni, Giacomo Mandelli, Claudia Rossi, Michela Vezzoli, Annamaria Aprile, Carsten Werner Lederer, Alessandro Ambrosi, Franck Chanut, Francesca Sanvito, Andrea Calabria, Valentina Poletti, Fulvio Mavilio, Eugenio Montini, Luigi Naldini, Patrizia Cristofori, Giuliana Ferrari

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 9-28 (2018)

Gene therapy clinical trials require rigorous non-clinical studies in the most relevant models to assess the benefit-to-risk ratio. To support the clinical development of gene therapy for β-thalassemia, we performed in vitro and in vivo studies for

Externí odkaz: https://doaj.org/article/fa7c8bf590fd4385824802dbcaeebd2f

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

Autor: Francesca Sanvito, Ylenia Paleari, Annamaria Aprile, Franck Chanut, Luigi Naldini, Fulvio Mavilio, Patrizia Cristofori, Claudia Rossi, Maria Rosa Lidonnici, Giuliana Ferrari, Giacomo Mandelli, Eugenio Montini, Andrea Calabria, Alessandro Ambrosi, Valentina Poletti, Francesca Tiboni, Michela Vezzoli, Carsten W. Lederer

Publikováno v: Mol Ther Methods Clin Dev
Mol Ther Methods Clin Dev, 2018, 11, pp.9-28. ⟨10.1016/j.omtm.2018.09.001⟩
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 11, Iss, Pp 9-28 (2018)
Molecular Therapy-Methods & Clinical Development

Gene therapy clinical trials require rigorous non-clinical studies in the most relevant models to assess the benefit-to-risk ratio. To support the clinical development of gene therapy for β-thalassemia, we performed in vitro and in vivo studies for

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d91676d09e4f1d206a3e4150b26efc2f
https://doi.org/10.1016/j.omtm.2018.09.001

Conditioning Regimens in Long-Term Pre-Clinical Studies to Support Development of

Autor: Franck J A, Chanut, Francesca, Sanvito, Giuliana, Ferrari, Ilaria, Visigalli, Nicola, Carriglio, Raisa Jofra, Hernandez, Rossana, Norata, Claudio, Doglioni, Luigi, Naldini, Patrizia, Cristofori

Publikováno v: Human gene therapy. 32(1-2)

Hematopoietic stem cell gene therapy has become a successful therapeutic strategy for some inherited genetic disorders. Pre-clinical toxicity studies performed to support the human clinical trials using viral-mediated gene transfer and autologous hem

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::dca360ef46130ee7f206a6c142972e1f
https://pubmed.ncbi.nlm.nih.gov/32883113

A Diagnostic Approach for Rodent Progressive Cardiomyopathy and Like Lesions in Toxicology Studies up to 28 Days in the Sprague Dawley Rat (Part 2 of 2)

Autor: Vinicius Carreira, Carie L Kimbrough, Abraham Nyska, Gail Pearse, Noel Dybdal, James R. Hailey, Niraj Tripathi, James D Fikes, Jan Klapwijk, Susan A. Elmore, Ingrid Brees, Beverly E. Maleeff, Kathleen Biddle, Brian R. Berridge, Martin Lamb, Rick Adler, Patrizia Cristofori, Bernard S. Buetow, Jean-Guy Bienvenu, Heather C. Workman, Daniel Morton, Heath C. Thomas, James R. Turk, Jerry F. Hardisty, David A Rehagen, George A. Parker

Publikováno v: Toxicologic Pathology. 45:1055-1066

To test the diagnostic approach described in part 1 of this article, 2 exercises were completed by pathologists from multiple companies/agencies. Pathologist’s examination of whole slide image (WSI) heart sections from rats using personal diagnosti

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::811e9dc96e9e43f43e523b82ea909b7d
https://doi.org/10.1177/0192623317743948

Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

Autor: Mauro Biffi, Daniela Cesana, Federica Moalli, Tongyao Liu, Matteo Iannacone, Douglas Drager, Patrizia Cristofori, Sara Bartolaccini, Fabio Russo, Andrea Raimondi, Alessio Cantore, Ilaria Visigalli, Robert T. Peters, Eugenio Montini, Andrea Calabria, Michela Milani, Susannah Patarroyo-White, Andrea Annoni, Eduard Ayuso, Luigi Naldini

Publikováno v: Science Translational Medicine
Science Translational Medicine, American Association for the Advancement of Science, 2019, 11 (493), pp.eaav7325. ⟨10.1126/scitranslmed.aav7325⟩
Science Translational Medicine, American Association for the Advancement of Science (AAAS), 2019, 11 (493), pp.eaav7325. ⟨10.1126/scitranslmed.aav7325⟩
Sci Transl Med

International audience; Liver-directed gene therapy for the coagulation disorder hemophilia showed safe and effective results in clinical trials using adeno-associated viral vectors to replace a functional coagulation factor, although some unmet need

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6649d598dd8d5d1cee1da1118caa1bc3
https://doi.org/10.1126/scitranslmed.aav7325