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

Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to Plerixafor alone

Autor: Laura Bellio, Bernhard Gentner, Laura Zanaboni, Matilde Zambelli, Annamaria Aprile, Elena Cassinerio, Sarah Marktel, Ylenia Paleari, Cristina Parisi, Fabio Ciceri, Antonello E. Spinelli, Marta Claudia Frittoli, Maria Domenica Cappellini, Giacomo Mandelli, Giuliana Ferrari, Maria Rosa Lidonnici

For decades, bone marrow (BM) has been the preferred source of hematopoietic stem and progenitor cells (HSPCs) for transplants following myeloablative conditioning. At present, mobilized peripheral blood stem cells are commonly used for transplantati

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bd84e50a253067a347f8952ea0a4aa1d

Human CD34+ Cells from Different Sources Disclose a Specific Stemness Signature

Autor: Maria Rosa Lidonnici, Marta Claudia Frittoli, Matilde Zambelli, Giuliana Ferrari, Giacomo Mandelli, Annamaria Aprile, Bernhard Gentner, Fabio Ciceri, Maria Domenica Cappellini, Ylenia Paleari, Sarah Marktel, Antonello E. Spinelli, Laura Zanaboni, Elena Cassinerio, Cristina Parisi, Laura Bellio

Publikováno v: Blood. 128:4709-4709

Over the past decades outcomes of clinical hematopoietic stem cell transplants have established a clear relationship between the sources of hematopoietic stem cells (HSCs) infused and their differential homing and engraftment properties. For a long t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::b85be25ecb1a60624ea9cd7399e6ef9a
https://doi.org/10.1182/blood.v128.22.4709.4709

Intrinsic Molecular Features of Human Hematopoietic Stem Cells from Different Sources Define Their Specific Functional Properties

Autor: Bernhard Gentner, Annamaria Aprile, Sarah Marktel, Laura Bellio, Ylenia Paleari, Marta Claudia Frittoli, Elena Cassinerio, Fabio Ciceri, Giacomo Mandelli, Laura Zanaboni, Maria Domenica Cappellini, Giuliana Ferrari, Maria Rosa Lidonnici

Publikováno v: Blood. 126:3069-3069

Over the past decades outcomes of clinical hematopoietic stem cell transplants have established a clear relationship between the sources of hematopoietic stem cells (HSCs) infused and their differential homing and engraftment properties. For a long t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::b9b9590a61eb644e42eb69d1e45309db
https://doi.org/10.1182/blood.v126.23.3069.3069