Cell and Gene Therapy

Track 4: Viral gene therapy

The viral life cycle can be separated into two temporally distinct phases: infection and replication. This is known as the viral vectorization process. A sufficient quantity of a therapeutic gene must be delivered into the target tissue without significant harm for gene therapy to be effective. Each viral vector system has a unique set of characteristics that influence the applications for which it is suitable for use in gene therapy. For some diseases—for instance, hereditary disorders—long-term expression from a tiny percentage of cells would be sufficient, whereas other illnesses would call for high-but-transient gene expression. Gene transfer may be necessary into a significant portion of the aberrant cells, for instance, in gene therapies intended to disrupt a viral infectious process or restrict the proliferation of cancer cells by reactivating inactivated tumour suppressor genes.