Center for Genome Research,
Department of Life Sciences,
Center for Genome Research, Department of Life Sciences
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Muscle-derived hematopoietic stem cells are hematopoietic in origin.
Proceedings of the National Academy of Sciences of the United States of America Feb, 2002 | Pubmed ID: 11830662
Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement.
Journal of virology Apr, 2002 | Pubmed ID: 11907239
Myogenic stem cells from the bone marrow: a therapeutic alternative for muscular dystrophy?
Neuromuscular disorders : NMD Oct, 2002 | Pubmed ID: 12206789
The choice of a suitable lentivirus vector: transcriptional targeting.
Methods in molecular biology (Clifton, N.J.) , 2003 | Pubmed ID: 12824618
The future of gene therapy.
Nature Feb, 2004 | Pubmed ID: 14985734
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
Stem cell plasticity: time for a reappraisal?
Haematologica Mar, 2005 | Pubmed ID: 15749669
Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor.
Human gene therapy May, 2005 | Pubmed ID: 15916484
Gene therapy approaches for epidermolysis bullosa.
Clinics in dermatology Jul-Aug, 2005 | Pubmed ID: 16023940
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
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
Gene therapy in combination with tissue engineering to treat epidermolysis bullosa.
Expert opinion on biological therapy Apr, 2006 | Pubmed ID: 16548763
Site-specific integration into the human genome: ready for clinical application?
Rejuvenation research , 2006 | Pubmed ID: 17105384
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.
Nature medicine Dec, 2006 | Pubmed ID: 17115047
Hot spots of retroviral integration in human CD34+ hematopoietic cells.
Blood Sep, 2007 | Pubmed ID: 17507662
C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cells.
The Journal of cell biology Jun, 2007 | Pubmed ID: 17562792
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
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
Role of CD34 antigen in myeloid differentiation of human hematopoietic progenitor cells.
Stem cells (Dayton, Ohio) Apr, 2008 | Pubmed ID: 18192237
Towards a gene therapy clinical trial for epidermolysis bullosa.
Reviews on recent clinical trials May, 2006 | Pubmed ID: 18473966
Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology.
EMBO reports Jul, 2008 | Pubmed ID: 18578029
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
PPARdelta is a ligand-dependent negative regulator of vitamin D3-induced monocyte differentiation.
Carcinogenesis Feb, 2009 | Pubmed ID: 19056929
Tracking gene-modified T cells in vivo.
Methods in molecular biology (Clifton, N.J.) , 2009 | Pubmed ID: 19110640
Transcription factor binding sites are genetic determinants of retroviral integration in the human genome.
PloS one , 2009 | Pubmed ID: 19238208
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
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
Comprehensive genomic access to vector integration in clinical gene therapy.
Nature medicine Dec, 2009 | Pubmed ID: 19966782
Gene therapy: back on track?
EMBO reports Feb, 2010 | Pubmed ID: 20118988
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.
EMBO molecular medicine Aug, 2010 | Pubmed ID: 20665635
High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.
Blood Dec, 2010 | Pubmed ID: 20864581
High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion.
PloS one , 2010 | Pubmed ID: 21203516
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
Site-specific integration by the adeno-associated virus rep protein.
Current gene therapy Oct, 2011 | Pubmed ID: 21827397
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
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
Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors.
PLoS computational biology Dec, 2011 | Pubmed ID: 22144885
The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene.
PloS one , 2011 | Pubmed ID: 22164220
Gene therapy of skin adhesion disorders (mini review).
Current pharmaceutical biotechnology Aug, 2012 | Pubmed ID: 22250710
Alternative splicing caused by lentiviral integration in the human genome.
Methods in enzymology , 2012 | Pubmed ID: 22365773
Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts.
The Journal of clinical investigation May, 2012 | Pubmed ID: 22523069
Gene therapies need new development models.
Nature Oct, 2012 | Pubmed ID: 23038429
IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.
Blood Jan, 2013 | Pubmed ID: 23160470
Mechanisms of retroviral integration and mutagenesis.
Human gene therapy Feb, 2013 | Pubmed ID: 23330935
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Molecular therapy : the journal of the American Society of Gene Therapy Feb, 2013 | Pubmed ID: 23369965
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
Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication.
Virology May, 2013 | Pubmed ID: 23523133
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
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
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
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.
Science translational medicine Jan, 2014 | Pubmed ID: 24452262
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
Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.
Genes May, 2014 | Pubmed ID: 24840152
Genomic analysis of Sleeping Beauty transposon integration in human somatic cells.
PloS one , 2014 | Pubmed ID: 25390293
Perspectives on best practices for gene therapy programs.
Human gene therapy Mar, 2015 | Pubmed ID: 25654329
A single epidermal stem cell strategy for safe ex vivo gene therapy.
EMBO molecular medicine Apr, 2015 | Pubmed ID: 25724200
Nuclear architecture dictates HIV-1 integration site selection.
Nature May, 2015 | Pubmed ID: 25731161
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
JAMA Apr, 2015 | Pubmed ID: 25898053
Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.
PloS one , 2015 | Pubmed ID: 25978676
Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation.
Stem cell reports Apr, 2016 | Pubmed ID: 27050947
Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment.
Scientific reports Apr, 2016 | Pubmed ID: 27095295
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
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
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