Custom-designed proteins as novel therapeutic tools? The case of arrestins

What is it about?

Proteins are multi-functional little machines. Genetic disorders are usually associated with changes in some functions of affected protein. Compensatory changes in interacting proteins can bring the situation closer to normal, and therefore serve as therapeutic strategy. In case of gain-of-function mutations, compensation appears to be the most straightforward approach. This approach has been tested in animals: in mice overactive arrestin partially compensated for excessive rhodopsin signaling. Although this approach is still in its infancy, targeted redesign of individual functions of proteins offers a promise of a completely new therapeutic toolbox with huge potential.

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Why is it important?

There are no viable approaches to treat genetic disorders caused by gain-of-function mutations in proteins.