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Publication
Evaluation of pEPI-1 and pEPito expression systems for gene transfer to the retina
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Abstract(s)
O objectivo de estudo do nosso laboratório é o desenvolvimento de vectores não virais para a terapia génica ocular. O principal objectivo da terapia génica é a introdução de material genético no interior das células. Em teoria, o veículo ideal para a terapia génica é aquele que consegue penetrar eficientemente a membrana celular e libertar o material genético, sem desencadear uma resposta imunológica agressiva.
In this work we have tested the efficiency of enhanced plasmids, based on pEPI-1 and pEPito backbones, for retinal gene expression. These plasmids contain a number of modifications to enhance the duration of gene expression, such as 1) a matrix attachment region sequence to permit replication as episomal particle, 2) lower CpG content, to avoid silencing of the plasmid and 3) different promoters, including a tissue-specific promoter, RPE65, for targeted expression for RPE cells.
In this work we have tested the efficiency of enhanced plasmids, based on pEPI-1 and pEPito backbones, for retinal gene expression. These plasmids contain a number of modifications to enhance the duration of gene expression, such as 1) a matrix attachment region sequence to permit replication as episomal particle, 2) lower CpG content, to avoid silencing of the plasmid and 3) different promoters, including a tissue-specific promoter, RPE65, for targeted expression for RPE cells.
Description
Dissertação de Mestrado, Ciências Biomédicas, Departamento de Ciências Biomédicas e Medicina, Universidade do Algarve, 2011
Keywords
Terapia génica Retina S/MAR Motivos CpG pEPito