Tissue specific optimised AAV plasmids

Viral vectors are a new class of biologics that help treat diseases caused by defective gene function/proteins (‘gene therapy’). More than 20 companies worldwide the majority of which originate in the United States are applying viral vectors to conduct clinical studies.

A key hurdle when applying viral vectors is to limit the transduction (gene transfer) to the appropriate cells of a specific tissue without affecting their surrounding environment. But, how can an expression system differentiate between tissue or even cell type? SIRION Biotech, in co-operation with University of Munich (LMU) and University of Cologne, has developed a line of viral vectors with specific promotors that are only active in a specific set of targeted cells to initiate the desired gene expression. Using this method, the gene of interest is only being expressed in the targeted tissue which is relevant for the desired therapy. This improves the effectiveness of the therapy and also addresses safety concerns by reducing the likelihood of side effects.

Recently the company announced a new line of cell specific AAV construction plasmids, controlling expression in brain and retinal sensory cells, liver, cardiac and skeletal muscle. They are based on the commonly used AAV 2 single strand serotype and contain a classical multiple-cloning-site (MCS) for easy, customised manipulation by the experimenter.

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