Gene Therapy

∞ generated and posted on 2022.01.25 ∞

Cloning into existing multicellular organisms, particularly for the sake of addressing metabolic insufficiencies.

In Gene Therapy new genes are inserted into the chromosomes of cells, and ideally defective genes are outright replaced with non-defective ones, all towards the goal of addressing medical conditions stemming from the carriage of the defective gene.

The key aspect distinguishing gene therapy from simple gene cloning is that with gene therapy there are multiple cellular targets for this cloning. By contrast, with gene cloning, typically it is sufficient to modify only a single cell, which through reproduction then goes to produce multiple clones.

The vector in gene therapy often are modified viruses. This is the case because, just as with gene cloning via transduction, the viruses are inherently capable of delivering DNA cargo to cells. Through genetic engineering, it is also possible to modify the tropism of viruses so that they target specific cells and tissues.

The process of gene therapy can take place either in vivo, which is as gene therapy typically is envisaged, or in vitro, which is applicable particularly to cells that can be removed from the body and then returned. The differences is one of safety, with in vitro efforts typically inherently safer than in vivo ones since with the latter the entire body rather than select cells are exposed to the gene therapy vector.

Note that technically DNA vaccines represent a form gene therapy, whether delivered using vectors or instead as naked DNA.