Abstract
Gene therapy can be defined as the introduction of genetic material into a particular cell or tissue type to alter the pattern of gene expression to produce a therapeutic effect. There are three critical components to consider in designing and optimizing a gene therapy strategy for the treatment of any disease: a vector to introduce the gene, a device and procedure for delivering the vector to the appropriate tissue or organ, and a therapeutic gene. Each component must be chosen or modified to best suit the specific characteristics of the particular disease of interest.
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