Fulvio Mavilio

Publications

• Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Apr, 2017  |   Pubmed ID: 28237839
• Efficacy and Biodistribution Analysis of Intracerebroventricular Administration of an Optimized ScAAV9-SMN1 Vector in a Mouse Model of Spinal Muscular Atrophy
  Molecular Therapy. Methods & Clinical Development. 2016  |   Pubmed ID: 27652289
• Transcriptional, Epigenetic and Retroviral Signatures Identify Regulatory Regions Involved in Hematopoietic Lineage Commitment
  Scientific Reports. Apr, 2016  |   Pubmed ID: 27095295
• Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation
  Stem Cell Reports. Apr, 2016  |   Pubmed ID: 27050947
• Genome-Wide Definition of Promoter and Enhancer Usage During Neural Induction of Human Embryonic Stem Cells
  PloS One. 2015  |   Pubmed ID: 25978676
• Outcomes Following Gene Therapy in Patients with Severe Wiskott-Aldrich Syndrome
  JAMA. Apr, 2015  |   Pubmed ID: 25898053
• Nuclear Architecture Dictates HIV-1 Integration Site Selection
  Nature. May, 2015  |   Pubmed ID: 25731161
• A Single Epidermal Stem Cell Strategy for Safe Ex Vivo Gene Therapy
  EMBO Molecular Medicine. Apr, 2015  |   Pubmed ID: 25724200
• Perspectives on Best Practices for Gene Therapy Programs
  Human Gene Therapy. Mar, 2015  |   Pubmed ID: 25654329
• Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells
  PloS One. 2014  |   Pubmed ID: 25390293
• Genome-wide Analysis of Alpharetroviral Integration in Human Hematopoietic Stem/progenitor Cells
  Genes. May, 2014  |   Pubmed ID: 24840152
• Repairing Without Cutting: a Safer Alternative to Gene Correction?
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Apr, 2014  |   Pubmed ID: 24691177
• Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy
  Science Translational Medicine. Jan, 2014  |   Pubmed ID: 24452262
• RD2-MolPack-Chim3, a Packaging Cell Line for Stable Production of Lentiviral Vectors for Anti-HIV Gene Therapy
  Human Gene Therapy Methods. Aug, 2013  |   Pubmed ID: 23767932
• Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Sep, 2013  |   Pubmed ID: 23760447
• Genotoxic Signature in Cord Blood Cells of Newborns Exposed in Utero to a Zidovudine-based Antiretroviral Combination
  The Journal of Infectious Diseases. Jul, 2013  |   Pubmed ID: 23559464
• Nup153 and Nup98 Bind the HIV-1 Core and Contribute to the Early Steps of HIV-1 Replication
  Virology. May, 2013  |   Pubmed ID: 23523133
• Deletion of the LTR Enhancer/promoter Has No Impact on the Integration Profile of MLV Vectors in Human Hematopoietic Progenitors
  PloS One. 2013  |   Pubmed ID: 23383272
• 번역 하는 게놈 혁명: Monogenic 질병에 대 한 국제 유전자 치료 컨소시엄에 대 한 필요 합니다
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Feb, 2013  |   Pubmed ID: 23369965
• Mechanisms of Retroviral Integration and Mutagenesis
  Human Gene Therapy. Feb, 2013  |   Pubmed ID: 23330935
• IL-7 and IL-15 Instruct the Generation of Human Memory Stem T Cells from Naive Precursors
  Blood. Jan, 2013  |   Pubmed ID: 23160470
• Gene Therapies Need New Development Models
  Nature. Oct, 2012  |   Pubmed ID: 23038429
• Lentiviral Vector Integration in the Human Genome Induces Alternative Splicing and Generates Aberrant Transcripts
  The Journal of Clinical Investigation. May, 2012  |   Pubmed ID: 22523069
• Alternative Splicing Caused by Lentiviral Integration in the Human Genome
  Methods in Enzymology. 2012  |   Pubmed ID: 22365773
• Gene Therapy of Skin Adhesion Disorders (mini Review)
  Current Pharmaceutical Biotechnology. Aug, 2012  |   Pubmed ID: 22250710
• Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Apr, 2012  |   Pubmed ID: 22068429
• The GATA1-HS2 Enhancer Allows Persistent and Position-independent Expression of a β-globin Transgene
  PloS One. 2011  |   Pubmed ID: 22164220
• Estimated Comparative Integration Hotspots Identify Different Behaviors of Retroviral Gene Transfer Vectors
  PLoS Computational Biology. Dec, 2011  |   Pubmed ID: 22144885
• Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Nov, 2011  |   Pubmed ID: 21862999
• Site-specific Integration by the Adeno-associated Virus Rep Protein
  Current Gene Therapy. Oct, 2011  |   Pubmed ID: 21827397
• Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Oct, 2011  |   Pubmed ID: 21750532
• High-definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells After Donor Lymphocyte Infusion
  PloS One. 2010  |   Pubmed ID: 21203516
• High-definition Mapping of Retroviral Integration Sites Identifies Active Regulatory Elements in Human Multipotent Hematopoietic Progenitors
  Blood. Dec, 2010  |   Pubmed ID: 20864581
• Correction of Beta-thalassemia Major by Gene Transfer in Haematopoietic Progenitors of Pediatric Patients
  EMBO Molecular Medicine. Aug, 2010  |   Pubmed ID: 20665635
• Gene Therapy: Back on Track?
  EMBO Reports. Feb, 2010  |   Pubmed ID: 20118988
• Comprehensive Genomic Access to Vector Integration in Clinical Gene Therapy
  Nature Medicine. Dec, 2009  |   Pubmed ID: 19966782
• Integration of Retroviral Vectors Induces Minor Changes in the Transcriptional Activity of T Cells from ADA-SCID Patients Treated with Gene Therapy
  Blood. Oct, 2009  |   Pubmed ID: 19652199
• Transcriptional Enhancers Induce Insertional Gene Deregulation Independently from the Vector Type and Design
  Molecular Therapy : the Journal of the American Society of Gene Therapy. May, 2009  |   Pubmed ID: 19293778
• Transcription Factor Binding Sites Are Genetic Determinants of Retroviral Integration in the Human Genome
  PloS One. 2009  |   Pubmed ID: 19238208
• Tracking Gene-modified T Cells in Vivo
  Methods in Molecular Biology (Clifton, N.J.). 2009  |   Pubmed ID: 19110640
• PPARdelta is a Ligand-dependent Negative Regulator of Vitamin D3-induced Monocyte Differentiation
  Carcinogenesis. Feb, 2009  |   Pubmed ID: 19056929
• Correction of Laminin-5 Deficiency in Human Epidermal Stem Cells by Transcriptionally Targeted Lentiviral Vectors
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Dec, 2008  |   Pubmed ID: 18813277
• Genetic Modification of Somatic Stem Cells. The Progress, Problems and Prospects of a New Therapeutic Technology
  EMBO Reports. Jul, 2008  |   Pubmed ID: 18578029
• Role of CD34 Antigen in Myeloid Differentiation of Human Hematopoietic Progenitor Cells
  Stem Cells (Dayton, Ohio). Apr, 2008  |   Pubmed ID: 18192237
• Multilineage Hematopoietic Reconstitution Without Clonal Selection in ADA-SCID Patients Treated with Stem Cell Gene Therapy
  The Journal of Clinical Investigation. Aug, 2007  |   Pubmed ID: 17671653
• Long-term Engraftment of Single Genetically Modified Human Epidermal Holoclones Enables Safety Pre-assessment of Cutaneous Gene Therapy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Sep, 2007  |   Pubmed ID: 17579576
• C/EBPdelta Regulates Cell Cycle and Self-renewal of Human Limbal Stem Cells
  The Journal of Cell Biology. Jun, 2007  |   Pubmed ID: 17562792
• Hot Spots of Retroviral Integration in Human CD34+ Hematopoietic Cells
  Blood. Sep, 2007  |   Pubmed ID: 17507662
• Towards a Gene Therapy Clinical Trial for Epidermolysis Bullosa
  Reviews on Recent Clinical Trials. May, 2006  |   Pubmed ID: 18473966
• Correction of Junctional Epidermolysis Bullosa by Transplantation of Genetically Modified Epidermal Stem Cells
  Nature Medicine. Dec, 2006  |   Pubmed ID: 17115047
• Site-specific Integration into the Human Genome: Ready for Clinical Application?
  Rejuvenation Research. 2006  |   Pubmed ID: 17105384
• Gene Therapy in Combination with Tissue Engineering to Treat Epidermolysis Bullosa
  Expert Opinion on Biological Therapy. Apr, 2006  |   Pubmed ID: 16548763
• Retroviral Vector Integration Deregulates Gene Expression but Has No Consequence on the Biology and Function of Transplanted T Cells
  Proceedings of the National Academy of Sciences of the United States of America. Jan, 2006  |   Pubmed ID: 16432223
• T Lymphocytes Transduced with a Lentiviral Vector Expressing F12-Vif Are Protected from HIV-1 Infection in an APOBEC3G-independent Manner
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Oct, 2005  |   Pubmed ID: 16039909
• Gene Therapy Approaches for Epidermolysis Bullosa
  Clinics in Dermatology. Jul-Aug, 2005  |   Pubmed ID: 16023940
• Competitive Engraftment of Hematopoietic Stem Cells Genetically Modified with a Truncated Erythropoietin Receptor
  Human Gene Therapy. May, 2005  |   Pubmed ID: 15916484
• Stem Cell Plasticity: Time for a Reappraisal?
  Haematologica. Mar, 2005  |   Pubmed ID: 15749669
• Site-specific Integration of Functional Transgenes into the Human Genome by Adeno/AAV Hybrid Vectors
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Oct, 2004  |   Pubmed ID: 15451450
• The Future of Gene Therapy
  Nature. Feb, 2004  |   Pubmed ID: 14985734
• The Choice of a Suitable Lentivirus Vector: Transcriptional Targeting
  Methods in Molecular Biology (Clifton, N.J.). 2003  |   Pubmed ID: 12824618
• Myogenic Stem Cells from the Bone Marrow: a Therapeutic Alternative for Muscular Dystrophy?
  Neuromuscular Disorders : NMD. Oct, 2002  |   Pubmed ID: 12206789
• Transcriptional Targeting of Lentiviral Vectors by Long Terminal Repeat Enhancer Replacement
  Journal of Virology. Apr, 2002  |   Pubmed ID: 11907239
• 조 혈 줄기 세포 근육 파생은 원점에 조 혈입니다
  Proceedings of the National Academy of Sciences of the United States of America. Feb, 2002  |   Pubmed ID: 11830662