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Guide to using AAV vectors in gene therapy

Drug Discovery World

This is largely due to their highly stable Inverted Terminal Repeat (ITR) regions, as this secondary structure is prone to deletions during propagation in bacteria and cannot be read by standard Sanger sequencing. Dr O’Hara also provided an AAV plasmid preparation case study. Why are those important? How is the titer determined?

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Guide to using AAV vectors in gene therapy

Drug Discovery World

This is largely due to their highly stable Inverted Terminal Repeat (ITR) regions, as this secondary structure is prone to deletions during propagation in bacteria and cannot be read by standard Sanger sequencing. The post Guide to using AAV vectors in gene therapy appeared first on Drug Discovery World (DDW). Why are those important?

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Advances in cell line development

Drug Discovery World

Cell line development is an essential underpinning of the drug development process, enabling teams to test, optimise and manufacture therapeutics at a commercial scale. For drug discovery and development, cell lines allow researchers to test the efficacy of therapeutics before moving into in vivo studies.

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Improving quality control for CAR T cell therapies

Drug Discovery World

The immune system typically identifies foreign entities such as bacteria and viruses and activates lymphocytes such as T cells to destroy them with overwhelming force. If the gene does not integrate into the genome, the cell will not become a CAR T cell. The challenge of manufacturing CAR T cells.