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But, since most shots happen in a muscle - think of the shots you get in your arm - the team needed to figure out how to get muscle cells to act more like liver cells in their production of AAT, and then how to get the AAT produced in the muscle to the lungs and liver where it is needed. AAT, for instance, is best made in the liver. However, not all of the body's cells are capable of making all the proteins the body needs. 'But until now we did not understand how well the AAT protein was processed within the muscle at biochemical level.' 'We have completed three clinical trials in which we inject AAV containing the normal version of the AAT gene into muscle to create a 'sustained release' of the protein in AAT deficient patients,' says Flotte. Flotte, a pioneer in gene therapies, has developed a way to use the harmless adeno-associated virus, or AAV, as a vehicle to deliver gene therapies.

Flotte, the Celia and Isaac Haidak Professor, executive deputy chancellor, provost and dean of the T.H. Funding for the research was provided by the Alpha-1 Foundation and the National Institutes of Health.

'This project is the result of more than a decade of collaboration, and runs the gamut from lab-based basic science to the bedside,' says Daniel Hebert, professor of biochemistry and molecular biology at UMass Amherst and one of the paper's co-authors.

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It's an enormously complex series of problems that requires a research team with expertise in molecular biology, cellular biology, protein folding and gene therapy, as well as cutting-edge research facilities in which to conduct the work, such as the Models to Medicine Center at UMass Amherst's Institute for Applied Life Sciences, which hosts the labs where much of the research was completed. Finally, that correctly folded protein needs to make its way from the cell to wherever in the body it is needed. Next, you have to ensure that once the body starts making the protein it had previously been missing, that protein is correctly folded into its proper, final shape - in the case of AAT, that shape looks like a loaded mousetrap ready to be sprung. To do so, you first need to get the right protein-producing gene into the body, typically via an intramuscular injection - a shot - and to the specific cells that make that protein. These diseases must be treated either by delivering the missing proteins to the body, or, even better, teaching the body to make the missing proteins for itself by introducing an intact copy of the specific protein-producing gene into the right cell's DNA.īut it is no easy task to teach the body to make a missing protein. Production of defective AAT or inadequate amounts of AAT can cause serious lung and liver diseases. It is now clear that there is an entire range of diseases, such as AAT deficiency, that arise when our own bodies and produce dysfunctional protein at the genetic level. A revolution in disease treatment has occurred in recent years.

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