Fulvio Mavilio

University of Modena and Reggio Emilia — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

69h-index20.0kcitations302works2.02yr avg
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Research Topics

Virus-based gene therapy research(113), CRISPR and Genetic Engineering(83), Hemoglobinopathies and Related Disorders(44), RNA Interference and Gene Delivery(41), CAR-T cell therapy research(30)

Publications301 total

Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Nature Communications·2025· 10 citedOpen Access
Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease
Molecular Genetics and Metabolism·2025
Tu1723 HEMATOPOIETIC STEM CELL GENE THERAPY AS A TREATMENT FOR SEVERE CROHN'S DISEASE ASSOCIATED WITH PATHOGENIC NOD2 GENETIC VARIANTS
Gastroenterology·2024
HEMATOPOIETIC STEM CELL GENE THERAPY AS A NOVEL THERAPEUTIC APPROACH FOR SEVERE CROHN’S DISEASE ASSOCIATED WITH NOD2-DEFICIENCY
Inflammatory Bowel Diseases·2024· 1 citedOpen Access
HEMATOPOIETIC STEM CELL GENE THERAPY AS A NOVEL THERAPEUTIC APPROACH FOR SEVERE CROHN’S DISEASE ASSOCIATED WITH NOD2-DEFICIENCY
Gastroenterology·2024· 1 citedOpen Access
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·2024Open Access
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
The Lancet Neurology·2023· 101 citedOpen Access
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·2023· 4 citedOpen Access
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
Molecular Therapy — Nucleic Acids·2023· 14 citedOpen Access
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
2022· 2 citedOpen Access
Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia
Frontiers in Neuroscience·2022· 15 citedOpen Access
Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity
EMBO Molecular Medicine·2021· 42 citedOpen Access
Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients
Blood·2021· 19 cited
Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies
Blood·2021· 3 cited
Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases
Viruses·2021· 89 citedOpen Access
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
Blood Advances·2021· 74 citedOpen Access
Clinical management of sickle cell liver disease in children and young adults
Archives of Disease in Childhood·2020· 26 citedOpen Access
AUTOPHAGIC MYOPATHIES / MYOFIBRILLAR MYOPATHIES / DISTAL MYOPATHIES / POMPE DISEASE
Neuromuscular Disorders·2020
ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment (1053)
Neurology·2020· 10 cited
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
Science Advances·2020· 131 citedOpen Access
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Frequent Co-authors

Giuliana Ferrari(14), Claudio Bordignon(8), Alessandra Recchia(7), Antonio Simeone(7), Chiara Bonini(6), Dario Acampora(6), Daniela Sartori(6), Annarita Miccio(6), Vincenzo Zappavigna(5), Michele De Luca(4), Edoardo Boncinelli(4), Adrian J. Thrasher(4), Marina Cavazzana(4), G Russo(4), C Peschle(4), Claudia Cattoglio(4), Alessandro Aiuti(4), Anna Stornaiuolo(3), Giulio Cossu(3), Paolo Servida(3)