Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases

by Valentina Poletti, Fulvio Mavilio
Published 2021-08-01

Interactions between Retroviruses and the Host Cell Genome

by Valentina Poletti, Fulvio Mavilio
Published 2018-03-01

The Pharmacology of Gene and Cell Therapy

by Thomas J. Conlon, Fulvio Mavilio
Published 2018-03-01

Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

by Araksya Izmiryan, Clarisse Ganier, Matteo Bovolenta, Alain Schmitt, Fulvio Mavilio, Alain Hovnanian
Published 2018-09-01

The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene.

by Annarita Miccio, Valentina Poletti, Francesca Tiboni, Claudia Rossi, Antonella Antonelli, Fulvio Mavilio, Giuliana Ferrari
Published 2011-01-01

Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia

by Elizabeth Mercado-Ayón, Nathan Warren, Sarah Halawani, Layne N. Rodden, Lucie Ngaba, Yi Na Dong, Joshua C. Chang, Carlos Fonck, Fulvio Mavilio, Fulvio Mavilio, David R. Lynch, David R. Lynch, Hong Lin
Published 2022-03-01

Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors.

by Alessandro Ambrosi, Ingrid K Glad, Danilo Pellin, Claudia Cattoglio, Fulvio Mavilio, Clelia Di Serio, Arnoldo Frigessi
Published 2011-12-01

Correction: Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells.

by Giandomenico Turchiano, Maria Carmela Latella, Andreas Gogol Döring, Claudia Cattoglio, Fulvio Mavilio, Zsuzsanna Izsvák, Zoltán Ivics, Alessandra Recchia
Published 2020-01-01

Transcription factor binding sites are genetic determinants of retroviral integration in the human genome.

by Barbara Felice, Claudia Cattoglio, Davide Cittaro, Anna Testa, Annarita Miccio, Giuliana Ferrari, Lucilla Luzi, Alessandra Recchia, Fulvio Mavilio
Published 2009-01-01

Genome-Wide Analysis of Alpharetroviral Integration in Human Hematopoietic Stem/Progenitor Cells

by Arianna Moiani, Julia Debora Suerth, Francesco Gandolfi, Ermanno Rizzi, Marco Severgnini, Gianluca De Bellis, Axel Schambach, Fulvio Mavilio
Published 2014-05-01

Genomic analysis of Sleeping Beauty transposon integration in human somatic cells.

by Giandomenico Turchiano, Maria Carmela Latella, Andreas Gogol-Döring, Claudia Cattoglio, Fulvio Mavilio, Zsuzsanna Izsvák, Zoltán Ivics, Alessandra Recchia
Published 2014-01-01

Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors.

by Arianna Moiani, Annarita Miccio, Ermanno Rizzi, Marco Severgnini, Danilo Pellin, Julia Debora Suerth, Christopher Baum, Gianluca De Bellis, Fulvio Mavilio
Published 2013-01-01

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

by Annalisa Lattanzi, Stephanie Duguez, Arianna Moiani, Araksya Izmiryan, Elena Barbon, Samia Martin, Kamel Mamchaoui, Vincent Mouly, Francesco Bernardi, Fulvio Mavilio, Matteo Bovolenta
Published 2017-06-01

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

by Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio
Published 2018-06-01

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation

by Alessia Cavazza, Annarita Miccio, Oriana Romano, Luca Petiti, Guidantonio Malagoli Tagliazucchi, Clelia Peano, Marco Severgnini, Ermanno Rizzi, Gianluca De Bellis, Silvio Bicciato, Fulvio Mavilio
Published 2016-04-01

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

by Valentina Poletti, Fabrizia Urbinati, Sabine Charrier, Guillaume Corre, Roger P. Hollis, Beatriz Campo Fernandez, Samia Martin, Michael Rothe, Axel Schambach, Donald B. Kohn, Fulvio Mavilio
Published 2018-12-01

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

by Nicole Armbruster, Annalisa Lattanzi, Matthieu Jeavons, Laetitia Van Wittenberghe, Bernard Gjata, Thibaut Marais, Samia Martin, Alban Vignaud, Thomas Voit, Fulvio Mavilio, Martine Barkats, Ana Buj-Bello
Published 2016-01-01

Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

by Michelle Eggers, Charles H Vannoy, Jianyong Huang, Pravinkumar Purushothaman, Jacqueline Brassard, Carlos Fonck, Hui Meng, Mariah J Prom, Michael W Lawlor, Justine Cunningham, Chanchal Sadhu, Fulvio Mavilio
Published 2022-01-01

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

by Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano, Tristan Felix, Valentina Poletti, Samantha Scaramuzza, Sophie Ramadier, Cecile Masson, Giuliana Ferrari, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio
Published 2023-06-01

Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.

by Valentina Poletti, Alessia Delli Carri, Guidantonio Malagoli Tagliazucchi, Andrea Faedo, Luca Petiti, Emilia Maria Cristina Mazza, Clelia Peano, Gianluca De Bellis, Silvio Bicciato, Annarita Miccio, Elena Cattaneo, Fulvio Mavilio
Published 2015-01-01