Search results for " gene therapy"

showing 4 items of 24 documents

DEVELOPMENT OF S/MAR MINICIRLES VECTOR FOR PERSISTENT EXPRESSION IN VIVO.

2009

An ideal vector for gene therapy must fulfil the following requirements: non-toxicity, mitotic stability and persistent therapeutic levels of transgene expression. Viral vectors are widely used due to their ability to sustain prolonged expression. Their potentially tumorigenic effects are a limiting factor for in vivo applications. Non-viral vectors, which can be designed to be free from viral sequences, are a promising alternative for gene transfer although they often produce transient transgene expression. This limitation of this vector type is primarily due to bacterial sequences they contain and these have proven to be toxic for the mammalian cells as they contain a high number of unmet…

Settore MED/38 - Pediatria Generale E SpecialisticaMINICIRCLE VECTOR NON-VIRAL GENE THERAPY.
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Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver.

2010

We have previously described the development of a scaffold/matrix attachment region (S/MAR) episomal vector system for in vivo application and demonstrated its utility to sustain transgene expression in the mouse liver for at least 6 months following a single administration. Subsequently, we observed that transgene expression is sustained for the lifetime of the animal. The level of expression, however, does drop appreciably over time. We hypothesised that by eliminating the bacterial components in our vectors, we could improve their performance since bacterial sequences have been shown to be responsible for the immunotoxicity of the vector and the silencing of its expression when applied i…

TransgeneGenetic VectorsEnzyme-Linked Immunosorbent AssayBiologyMinicircleMolecular biologyPolymerase Chain ReactionScaffold/matrix attachment region (S/MAR) – Minicircle – Plasmid – Non-viral – Gene therapy – Liver – Hydrodynamic deliveryBlotting SouthernMicePlasmidSettore MED/38 - Pediatria Generale E SpecialisticaLiverIn vivoCell Line TumorDrug DiscoveryGene expressionMolecular MedicineGene silencingAnimalsHumansExpression cassetteTransgenesScaffold/matrix attachment regionGenetics (clinical)Journal of molecular medicine (Berlin, Germany)
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Hematopoietic Stem Cell Mobilization for Gene Therapy: The Combination of G-CSF+Plerixafor in Patients with Beta-Thalassemia Major Provides High Yiel…

2015

Abstract Hematopoietic stem cell engineering is a promising therapy to cure b-thalassemia, in particular for patients who lack a suitable BM donor for allogeneic transplantation. Since the engrafted gene-corrected stem cells will not have any selective advantage over the unmodified ones, the effectiveness of the therapy in this setting largely depends on the infusion of high numbers of gene-modified cells and on the conditioning regimen. The quality of the infused cells is also crucial for the clinical outcome and the duration of the therapeutic effect. HSPCs mobilization, particularly when G-CSF and plerixafor are used in combination, has been proved to be the optimal approach to harvest a…

business.industryPlerixaforImmunologyHematopoietic stem cellHematopoietic Stem Cell Mobilization Gene Therapy Beta-Thalassemia.Cell BiologyHematologyLeukapheresisCD38PharmacologyBiochemistryCXCR4Granulocyte colony-stimulating factorSettore BIO/18 - Geneticamedicine.anatomical_structureImmunologyMedicineStem cellbusinessHematopoietic Stem Cell Mobilizationmedicine.drug
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Effect of composition of Solid Lipid Nanoparticles on their chemical-physical properties and potential for gene therapy

2015

nanoparticles gene therapyLIPID NANOPARTICLESGENE THERAPYSURFACTANT
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