Search results for "Minicircle"

showing 3 items of 3 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.
researchProduct

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)
researchProduct

Development of a new S/MAR containing Minicircle DNA vector: a new promise for Gene Therapy.

2009

A barrier limiting the use of nonviral vectors for gene therapy is related to the short duration of transgene expression in vivo. Development, evaluation, and optimisation of a long term transgene expression using a non viral vector is currently the primary aim in our research field. Recently, we have demonstrate that a nonviral episomal plasmid (pDNA) vector combined with a scaffold/matrix attachment region (S/MAR) is able to sustain long-term expression in murine liver for at least six months following hydrodynamic injection. However, plasmids contain sequences, which are essential for propagation in bacteria but are unnecessary for expression in mammalian cells. These bacterial component…

Settore MED/38 - Pediatria Generale E Specialistica“Minicircle VECTOR" S/MAR.
researchProduct