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  1. Pubblicazioni

HUMAN GENE THERAPY

Rivista
Codice:
E078680
ISSN:
1043-0342
  • Dati Generali

Dati Generali

Pubblicazioni (24)

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  • decrescente
A Retroviral Vector Containing a Muscle-Specific Enhancer Drives Gene Expression Only in Differentiated Muscle Fibers
Articolo
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells
Articolo
Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor
Articolo
Construction and in vitro functional evaluation of a low-density lipoprotein receptor/transferrin fusion protein as a therapeutic tool for familial hypercholesterolemia
Articolo
Corrective transduction of human epidermal stem cells in laminin-5-dependent Junctional Epidermolysis Bullosa.
Articolo
Effective retrovirus-mediated gene transfer in normal and mutant human melanocytes
Articolo
Gene Editing for the Treatment of Primary Immunodeficiency Diseases
Articolo
Gene Therapy for Hemoglobinopathies
Articolo
Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study
Articolo
In vitro and in vivo CRISPR/Cas9-mediated genome editing to downregulate dominant mutations in Rhodopsin gene
Poster
Introductory remarks
Articolo
Low-density lipoprotein (LDL) receptor/transferrin fusion protein: in vivoproduction and functional evaluation as a potential therapeutic tool forlowering plasma LDL cholesterol.
Articolo
Mechanisms of retroviral integration and mutagenesis
Articolo
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67phox-Deficient Chronic Granulomatous Disease
Articolo
Non viral gene transfer via Sleeping beauty transposon for Collagen VII delivery in human primary keratinocytes.
Poster
Perspectives on Best Practices for Gene Therapy Programs
Articolo
Retroviral vector-mediated gene transfer into human primary myogenic cells leads to expression in muscle fibers in vivo
Articolo
Reversible immortalization of human myogenic cells by site-specific excision of a retrovitally transferred oncogene
Articolo
Surrounding pancreatic adenocarcinoma by killer mesenchymal stromal/stem cells
Articolo
Targeted integration of a large transgene cassette by TALEN and ZFN-mediated homologous recombination.
Poster
The regulation of differentiation in mesenchymal stem cells
Articolo
Toward epidermal stem cell-mediated ex vivo gene therapy of junctional epidermolysis bullosa
Articolo
Transduction of human hematopoietic stem cells by lentiviral vectors pseudotyped with the RD114-TR chimeric envelope glycoprotein
Articolo
Transfer of the HSV-tk Gene into Donor Peripheral Blood Lymphocytes for In Vivo Modulation of Donor Anti Tumor Immunity after Allogeneic Bone Marrow Transplantation. The San Raffaele Hospital, Milan, Italy
Articolo
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