Fulvio Mavilio

Muscle-derived hematopoietic stem cells are hematopoietic in origin.

Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement.

Myogenic stem cells from the bone marrow: a therapeutic alternative for muscular dystrophy?

The choice of a suitable lentivirus vector: transcriptional targeting.

The future of gene therapy.

Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors.

Stem cell plasticity: time for a reappraisal?

Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor.

Gene therapy approaches for epidermolysis bullosa.

T Lymphocytes transduced with a lentiviral vector expressing F12-Vif are protected from HIV-1 infection in an APOBEC3G-independent manner.

Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells.

Gene therapy in combination with tissue engineering to treat epidermolysis bullosa.

Site-specific integration into the human genome: ready for clinical application?

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.

Hot spots of retroviral integration in human CD34+ hematopoietic cells.

C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cells.

Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy.

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.

Role of CD34 antigen in myeloid differentiation of human hematopoietic progenitor cells.

Towards a gene therapy clinical trial for epidermolysis bullosa.

Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology.

Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectors.

PPARdelta is a ligand-dependent negative regulator of vitamin D3-induced monocyte differentiation.

Tracking gene-modified T cells in vivo.

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

Transcriptional enhancers induce insertional gene deregulation independently from the vector type and design.

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Comprehensive genomic access to vector integration in clinical gene therapy.

Gene therapy: back on track?

Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion.

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.

Site-specific integration by the adeno-associated virus rep protein.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.

Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.

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

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

Gene therapy of skin adhesion disorders (mini review).

Alternative splicing caused by lentiviral integration in the human genome.

Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts.

Gene therapies need new development models.

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.

Mechanisms of retroviral integration and mutagenesis.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

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

Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication.

Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination.

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.

RD2-MolPack-Chim3, a packaging cell line for stable production of lentiviral vectors for anti-HIV gene therapy.

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Repairing without cutting: a safer alternative to gene correction?

Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.

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

Perspectives on best practices for gene therapy programs.

A single epidermal stem cell strategy for safe ex vivo gene therapy.

Nuclear architecture dictates HIV-1 integration site selection.

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation.

Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment.

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

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.