Genes and drugs

Some medical drugs work by turning on a which can compensate for the defective activity of another gene. student Daniel Lai explains how this might offer a potential treatment for some patients with muscular dystrophy (where dystrophin is not made)?

Daniel Lai (PhD student, Auckland University): In some rare cases with dystrophin problems, utrophin [a related ] seems to come up and take its place enough to lessen the effects [it fulfils the role of the absent dystrophin, lessening the effects of DMD disease].

So you try and bring it up [by increasing its concentration in the ]. You don’t have to do a genetic manipulation to bring it up. You can follow a chemical approach, a pharmacological approach, like you add some drug and it’ll bring up the expression of this [the utrophin gene]. And this gene, let’s say B [utrophin], may be able to partially take the place of gene A [dystrophin, which is not expressed in DMD patients]. So you’re not fully recovering from the disease but you’re lessening the effects of it; it’s not as bad.