Fulvio MAVILIO
Università degli Studi di Modena e Reggio Emilia
H-index: 76
Europe-Italy
Top articles of Fulvio MAVILIO
Title | Journal | Author(s) | Publication Date |
|---|---|---|---|
OP29 Haematopoietic stem cell gene therapy as a treatment for severe Crohn’s Disease associated with pathogenic NOD2 genetic variants | Journal of Crohn's and Colitis | F Enjalbert M del Mar Masdeu T Zabinski JS Narean S Elavazhagan | 2024/1/1 |
HEMATOPOIETIC STEM CELL GENE THERAPY AS A NOVEL THERAPEUTIC APPROACH FOR SEVERE CROHN’S DISEASE ASSOCIATED WITH NOD2-DEFICIENCY | Inflammatory Bowel Diseases | Florence Enjalbert Maria Del Mar Masdeu Tomasz Zabinski Janakan Sam Narean Christopher Whiting | 2024/2/1 |
Bifunctional lentiviral vectors allowing the bs-globin silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of sickle cell disease | 2023/11/9 | ||
High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy | The American Journal of Human Genetics | Gaia Andreoletti Oriana Romano Hsin-Jung Chou Mahjoubeh J Sefid-Dashti Andrea Grilli | 2023/10/5 |
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial | The Lancet Neurology | Perry B Shieh Nancy L Kuntz James J Dowling Wolfgang Müller-Felber Carsten G Bönnemann | 2023/12/1 |
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies | Molecular Therapy-Nucleic Acids | Mégane Brusson Anne Chalumeau Pierre Martinucci Oriana Romano Tristan Felix | 2023/6/13 |
Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity | EMBO Molecular Medicine | Michelle Eggers Charles H Vannoy Jianyong Huang Pravinkumar Purushothaman Jacqueline Brassard | 2022/1/11 |
Cerebellar pathology in an inducible mouse model of Friedreich ataxia | Frontiers in Neuroscience | Elizabeth Mercado-Ayón Nathan Warren Sarah Halawani Layne N Rodden Lucie Ngaba | 2022/3/24 |
Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b-hemoglobinopathies | 2022/5/26 | ||
Clinical management of sickle cell liver disease in children and young adults | Eirini Kyrana David Rees Florence Lacaille Emer Fitzpatrick Mark Davenport | 2021/4/1 | |
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells | Blood advances | Giulia Pavani Anna Fabiano Marine Laurent Fatima Amor Erika Cantelli | 2021/3/9 |
Designing lentiviral vectors for gene therapy of genetic diseases | Valentina Poletti Fulvio Mavilio | 2021/8/2 | |
Clinical results of the Drepaglobe trial for sickle cell disease patients | Blood | Elisa Magrin Alessandra Magnani Michaela Semeraro Nicolas Hebert Laure Joseph | 2021/11/23 |
Stable integration of sin tranfer vectors | 2020/5/28 | ||
Correction: Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells | Plos one | Giandomenico Turchiano Maria Carmela Latella Andreas Gogol Döring Claudia Cattoglio Fulvio Mavilio | 2020/1/30 |
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype | Science advances | Leslie Weber Giacomo Frati Tristan Felix Giulia Hardouin Antonio Casini | 2020/2/12 |
ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment | Perry B Shieh Nancy Kuntz Barbara Smith James Dowling Wolfgang Müller-Felber | 2020/4/14 | |
Pre-clinical safety and efficacy findings of AT845, a novel gene replacement therapy for Pompe disease targeting skeletal muscle and heart | Molecular Genetics and Metabolism | Fulvio Mavilio Justine Cunningham Michelle Eggers Garrett Heffner Tim Stinchcombe | 2020/2/1 |
AUTOPHAGIC MYOPATHIES/MYOFIBRILLAR MYOPATHIES/DISTAL MYOPATHIES/POMPE DISEASE: P. 01 Pre-clinical safety and efficacy of AT845, a muscle-directed AAV-based gene replacement … | Neuromuscular Disorders | M Eggers C Vannoy J Huang P Purushottoman J Brassard | 2020/10/1 |
Editing a g-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype | Sci. Adv | Leslie Weber Giacomo Frati Tristan Felix Giulia Hardouin Antonio Casini | 2020 |
