Bibliotecas PUC-SP - Busca Integrada

Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases

af Valentina Poletti, Fulvio Mavilio
Udgivet i Viruses (2021-08-01)

Interactions between Retroviruses and the Host Cell Genome

af Valentina Poletti, Fulvio Mavilio
Udgivet i Molecular Therapy: Methods & Clinical Development (2018-03-01)

The Pharmacology of Gene and Cell Therapy

af Thomas J. Conlon, Fulvio Mavilio
Udgivet i Molecular Therapy: Methods & Clinical Development (2018-03-01)

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

af Araksya Izmiryan, Clarisse Ganier, Matteo Bovolenta, Alain Schmitt, Fulvio Mavilio, Alain Hovnanian
Udgivet i Molecular Therapy: Nucleic Acids (2018-09-01)

Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia

af 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
Udgivet i Frontiers in Neuroscience (2022-03-01)

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

af Annarita Miccio, Valentina Poletti, Valentina Poletti, Francesca Tiboni, Claudia Rossi, Antonella Antonelli, Fulvio Mavilio, Giuliana Ferrari
Udgivet i PLoS ONE (2011-01-01)

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

af Alessandro Ambrosi, Ingrid K Glad, Danilo Pellin, Claudia Cattoglio, Fulvio Mavilio, Clelia Di Serio, Arnoldo Frigessi
Udgivet i PLoS Computational Biology (2011-12-01)

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

af Giandomenico Turchiano, Maria Carmela Latella, Andreas Gogol Döring, Claudia Cattoglio, Fulvio Mavilio, Zsuzsanna Izsvák, Zoltán Ivics, Alessandra Recchia
Udgivet i PLoS ONE (2020-01-01)

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

af Arianna Moiani, Julia Debora Suerth, Francesco Gandolfi, Ermanno Rizzi, Marco Severgnini, Gianluca De Bellis, Axel Schambach, Fulvio Mavilio
Udgivet i Genes (2014-05-01)

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

af Giandomenico Turchiano, Maria Carmela Latella, Andreas Gogol-Döring, Claudia Cattoglio, Fulvio Mavilio, Zsuzsanna Izsvák, Zoltán Ivics, Alessandra Recchia
Udgivet i PLoS ONE (2014-01-01)

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

af Barbara Felice, Claudia Cattoglio, Davide Cittaro, Anna Testa, Annarita Miccio, Giuliana Ferrari, Lucilla Luzi, Alessandra Recchia, Fulvio Mavilio
Udgivet i PLoS ONE (2009-01-01)

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

af Arianna Moiani, Annarita Miccio, Ermanno Rizzi, Marco Severgnini, Danilo Pellin, Julia Debora Suerth, Christopher Baum, Gianluca De Bellis, Fulvio Mavilio
Udgivet i PLoS ONE (2013-01-01)

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

af Annalisa Lattanzi, Stephanie Duguez, Arianna Moiani, Araksya Izmiryan, Elena Barbon, Samia Martin, Kamel Mamchaoui, Vincent Mouly, Francesco Bernardi, Fulvio Mavilio, Matteo Bovolenta
Udgivet i Molecular Therapy: Nucleic Acids (2017-06-01)

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

af Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio
Udgivet i Molecular Therapy: Methods & Clinical Development (2018-06-01)

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation

af Alessia Cavazza, Annarita Miccio, Oriana Romano, Luca Petiti, Guidantonio Malagoli Tagliazucchi, Clelia Peano, Marco Severgnini, Ermanno Rizzi, Gianluca De Bellis, Silvio Bicciato, Fulvio Mavilio
Udgivet i Stem Cell Reports (2016-04-01)

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

af 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
Udgivet i Molecular Therapy: Methods & Clinical Development (2018-12-01)

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

af 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
Udgivet i Molecular Therapy: Methods & Clinical Development (2016-01-01)

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

af 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
Udgivet i EMBO Molecular Medicine (2021-12-01)

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency

af Tayebeh Soheili, Amandine Durand, Fernando E. Sepulveda, Julie Rivière, Chantal Lagresle-Peyrou, Hanem Sadek, Geneviève de Saint Basile, Samia Martin, Fulvio Mavilio, Marina Cavazzana, Isabelle André-Schmutz
Udgivet i Blood Advances (2017-12-01)

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

af 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
Udgivet i Molecular Therapy: Nucleic Acids (2023-06-01)