Could the protein dystrophin be artificially synthesised and if so could patients with DMD (Duchenne Muscular Dystrophy) benefit from it?

//Now I don't have much scientific background other than a high school biology qualification

I met someone the other day the same age as me who suffers from DMD, so I did some research and curiosity led to this question.

Sorry if I used the wrong tags or/and this question isn't allowed. \


Great question.

Yes, it is almost certainly possible to artificially synthesize just about any protein. There can be difficulties with certain proteins, but these are more engineering problems rather than true limits to artificial synthesis.

The problem is getting the protein where it needs to be to be functional!

A great comparison is insulin. Insulin is an important regulatory protein that floats around in the bloodstream. That makes insulin therapy a relatively straightforward solution to a problem of not enough insulin: all you have to do is make or isolate some insulin and inject it into the bloodstream.

Dystrophin, however, is a structural protein that acts inside cells. Proteins are really big, and they can't be simply taken up by cells. There's no mechanism to get an arbitrary protein inside: any proteins that can be taken up by cells need to do so via some specialized mechanism.

If you wanted to replace a defective allele for dystrophin, then, the most feasible approach would be gene therapy: getting a patient's own cells to produce a functional copy of the protein. Efforts to entirely or partially replace dystrophin via gene therapy are a current area of research, but there are still many challenges to gene therapy approaches in general.


Harper, S. Q., Hauser, M. A., DelloRusso, C., Duan, D., Crawford, R. W., Phelps, S. F., ... & Chamberlain, J. S. (2002). Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nature medicine, 8(3), 253.

McGreevy, J. W., Hakim, C. H., McIntosh, M. A., & Duan, D. (2015). Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy. Disease models & mechanisms, 8(3), 195-213.

Pichavant, C., Aartsma-Rus, A., Clemens, P. R., Davies, K. E., Dickson, G., Takeda, S. I., ... & Tremblay, J. P. (2011). Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Molecular Therapy, 19(5), 830-840.

Verhaart, I. E., & Aartsma-Rus, A. (2012). Gene therapy for Duchenne muscular dystrophy. Current opinion in neurology, 25(5), 588-596.


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