Research Topics
Species | Fulvio MavilioSummaryAffiliation: University of Modena and Reggio Emilia Country: Italy Publications
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Detail Information
Publications
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cellsFulvio Mavilio
Department of Biomedical Sciences, University of Modena and Reggio Emilia, Via Campi 287, 41100 Modena, Italy
Nat Med 12:1397-402. 2006..These data show that ex vivo gene therapy of JEB is feasible and leads to full functional correction of the disease...
Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technologyFulvio Mavilio
Molecular Biology and Centre for Regenerative Medicine at University of Modena and Reggio Emilia, Modena, Italy
EMBO Rep 9:S64-9. 2008
Site-specific integration by the adeno-associated virus rep proteinAlessandra Recchia
Center for Regenerative Medicine, University of Modena and Reggio Emilia, Via Gottardi 100, Modena, Italy
Curr Gene Ther 11:399-405. 2011..This region (AAVS1) has characteristics that make it an ideal target for somatic transgenesis...
Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectorsFrancesca Di Nunzio
Department of Biomedical Sciences, University of Modena and Reggio Emilia, Modena, Italy
Mol Ther 16:1977-85. 2008..These vectors are therefore an effective, and potentially more safe, alternative to MLV-based retroviral vectors in gene therapy of JEB.Molecular Therapy (2008) 16 12, 1977-1985 doi:10.1038/mt.2008.204...
PPARdelta is a ligand-dependent negative regulator of vitamin D3-induced monocyte differentiationAthina Lymboussaki
Department of Biomedical Sciences, University of Modena and Reggio Emilia, 41100 Modena, Italy
Carcinogenesis 30:230-7. 2009....
Transcriptional enhancers induce insertional gene deregulation independently from the vector type and designGiulietta Maruggi
Department of Biomedical Sciences, University of Modena and Reggio Emilia, Modena, Italy
Mol Ther 17:851-6. 2009..This study indicates that insertional gene activation is determined by the characteristics of the transcriptional regulatory elements carried by the vector, and is largely independent from the vector type or design...
Role of CD34 antigen in myeloid differentiation of human hematopoietic progenitor cellsSimona Salati
Department of Biomedical Sciences, Biological Chemistry Section, University of Modena and Reggio Emilia, Via Campi 287, 41100 Modena, Italy
Stem Cells 26:950-9. 2008....
Tracking gene-modified T cells in vivoAlessandra Recchia
Department of Biomedical Sciences, University of Modena and Reggio Emilia, Modena, Italy
Methods Mol Biol 506:391-401. 2009..This chapter provides a description of these techniques and clues to their rational use in a clinical setting...
Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptorFabrizia Urbinati
Department of Biomedical Sciences, University of Modena and Reggio Emilia, 41100 Modena, Italy
Hum Gene Ther 16:594-608. 2005....
Gene therapy of skin adhesion disorders (mini review)Alessia Cavazza
Center for Regenerative Medicine, University of Modena and Reggio Emilia, Via Gottardi 100, 41125 Modena, Italy
Curr Pharm Biotechnol 13:1868-76. 2012..This paper critically reviews the progress and prospects of gene therapy for skin adhesion defects, and the factors currently limiting its development...
Site-specific integration into the human genome: ready for clinical application?Alessandra Recchia
Department of Biochemical Sciences, University of Modena and Reggio Emilia, Modena, Italy
Rejuvenation Res 9:446-9. 2006....
Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cellsAlessandra Recchia
Cancer Immunotherapy and Gene Therapy Program, and Bone Marrow Transplantation Unit, Istituto Scientifico H. San Raffaele, Via Olgettina 58, 20132 Milan, Italy
Proc Natl Acad Sci U S A 103:1457-62. 2006..Despite the potentially dangerous interactions with the T cell genome, retroviral integration has therefore little consequence on the safety and efficacy of T cell transplantation...
The future of gene therapyMarina Cavazzana-Calvo
Immunology and Pediatric Haematology Unit, Hospital Necker, 75743 Paris, Cedex 15, France
Nature 427:779-81. 2004
Towards a gene therapy clinical trial for epidermolysis bullosaStefano Ferrari
Epithelial Stem Cell Research Centre, Veneto Eye Bank Foundation, Ospedale Civile SS Giovanni e Paolo, Sestiere Castello 6777, 30122 Venezia, Italy
Rev Recent Clin Trials 1:155-62. 2006....
The choice of a suitable lentivirus vector: transcriptional targetingFrancesco Lotti
HSR-Telethon Institute of Gene Therapy, Istituto Scientifico H. San Raffaele, Milan, Italy
Methods Mol Biol 229:17-27. 2003
Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapyFernando Larcher
Epithelial Biomedicine Division, Centro de Investigaciones Energéticas Medioambientales y Tecnologicas, Centro de Investigación Biomédica en Red de Enfermedades, Raras, Madrid, Spain
Mol Ther 15:1670-6. 2007..Our results provide an experimental basis for previously untested assumptions about the in vivo behavior of epidermal stem cells prospectively isolated in vitro and pave the way for a safer approach to cutaneous gene therapy...
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapyAlessandro Aiuti
San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
J Clin Invest 117:2233-40. 2007..These data have important implications for the biology of retroviral vectors, the dynamics of genetically modified HSCs, and the safety of gene therapy...
C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cellsVanessa Barbaro
Epithelial Stem Cell Research Center, The Veneto Eye Bank Foundation, H SS Giovanni and Paolo, 30100 Venice, Italy
J Cell Biol 177:1037-49. 2007....
Hot spots of retroviral integration in human CD34+ hematopoietic cellsClaudia Cattoglio
Italian Institute of Technology, Unit of Molecular Neuroscience, Istituto Scientifico H San Raffaele, Milan, Italy
Blood 110:1770-8. 2007..The lower propensity of LV vectors for integrating in potentially dangerous regions of the human genome may be a factor determining a better safety profile for gene therapy applications...
Gene therapy in combination with tissue engineering to treat epidermolysis bullosaStefano Ferrari
Epithelial Stem Cell Research Centre, Veneto Eye Bank Foundation, Ospedale Civile SS Giovanni e Paolo, Sestiere Castello 6777, 30122 Venezia, Italy
Expert Opin Biol Ther 6:367-78. 2006..The progress made has led the authors' group to submit a request for a Phase I/II ex vivo therapy clinical trial for patients with junctional EB...
T Lymphocytes transduced with a lentiviral vector expressing F12-Vif are protected from HIV-1 infection in an APOBEC3G-independent mannerGiuliana Vallanti
Molmed SpA, Milan, Italy
Mol Ther 12:697-706. 2005..Surprisingly, however, the F12-Vif-mediated inhibition does not depend on the reestablishment of the AP3G function...
Gene therapy approaches for epidermolysis bullosaStefano Ferrari
Epithelial Stem Cell Research Centre, Veneto Eye Bank Foundation, 30122 Venice, Italy
Clin Dermatol 23:430-6. 2005..Gene modification of stem cells in combination with advanced tissue-engineering techniques could therefore represent a realistic option for patients with epidermolysis bullosa...
Stem cell plasticity: time for a reappraisal?Roberto M Lemoli
Istituto di Ematologia e Oncologia Medica L e A Seragnoli, Universita di Bologna, Bologna, Italia
Haematologica 90:360-81. 2005..In addition to the authors' own work, the present review examines articles published in journals covered by the Science Citation Index and Medline...
Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectorsAlessandra Recchia
Istituto Scientifico H. San Raffaele, 20132 Milan, Italy
Mol Ther 10:660-70. 2004..Nonrandom integration of double-stranded DNA can therefore be obtained ex vivo and in vivoby the use of hybrid Ad/AAV vectors, in the absence of toxicity and with efficiency compatible with gene therapy applications...
Myogenic stem cells from the bone marrow: a therapeutic alternative for muscular dystrophy?Giuliana Ferrari
TIGET, Istituto Scientifico H San Raffaele, Via Olgettina 58, 20132 Milan, Italy
Neuromuscul Disord 12:S7-10. 2002..Expansion and active recruitment to myogenic differentiation of transplanted haematopoietic cells are therefore critical factors for a future use of bone marrow transplantation in cell/gene therapy of muscular dystrophy...
Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacementFrancesco Lotti
TIGET, Istituto Scientifico H San Raffaele, 20132 Milan, Italy
J Virol 76:3996-4007. 2002....
