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Andrea Amalfitano, D.O., Ph.D.

Research

Our laboratory has focused its efforts upon determining the feasibility of gene transfer to cure human genetic and non-genetic diseases, and to eventually translate that knowledge into the clinical realm. Therefore, we are utilizing multiple modalities in our investigations, and we are studying a variety of disease models. For example, all gene therapies will rely upon a vector to "deliver" the required genetic information into an appropriate target cell. To address this need, we have developed a uniquely improved gene transfer vector, one that is derived from a simple cold virus, (adenovirus). Our publications demonstrate that this vector can safely and efficiently deliver genetic information into a variety of organs in a living individual. The vector we have developed also has had several important safety attributes designed into it, thus making its potential for safe use in patients highly likely. Please note our multiple publications highlighting our progress to date.

One important problem with introduction of genetic material into a living organism is triggering of the innate immune response. Our recent work has now begun to identify key components of the multi-faceted innate immune system, thusly guiding our future efforts to subvert these systems to allow for safer gene transfer.

As we develop our vector technologies, and address the toxicities associated with gene transfer, we have also begun to study the use of these vectors in a variety of clinically important situations. The applications are numerous, thus we have created a number of vectors not only for our use, but also available for use by a number of investigators throughout Michigan State University, as well as for other national/international institutions or corporations. These studies are aimed at determining the ability of gene transfer to cure diseases affecting the liver, eye, hearing apparatus, muscles, heart, coronary arteries, as well as its ability to improve certain aspects of cancer therapies.

Intriguingly, we have recently begun to explore the potential of gene transfer to elicit beneficial immune responses in cancer patients. These studies will shed light on host-vector adaptive immune responses, and reveal how use of gene transfer can augment or minimize adaptive immune responses for beneficial clinical usage.

Finally, our laboratory has been pioneering research efforts into the potential for gene therapy to treat one of the most devastating forms of muscular dystrophy, known as Pompe disease. Pompe disease is a musculoskeletal and cardiac disease, that in it's most severe form rapidly kills babies in the first years of life. Young children, teenagers, as well as adults can also be affected by Pompe disease, resulting in wheelchair dependence, respiratory failure and death. In 1999 we were the first group to demonstrate that a single injection of our modified gene transfer vector into animal models of Pompe disease could result in a rapid reversal of pathology in ALL muscles of the animal. Our subsequent publications demonstrate that the approach is a long-lasting one. Cumulatively, our studies demonstrate that this form of muscular dystrophy may be among the first diseases to potentially benefit from the promise that gene "therapy" has always held out. Current issues that remain include minimizing the toxicity of such an approach, and developing the therapy into one that is practical for biotechnology corporations to pursue.

In summary, our research goals have been, and continue to be, focused upon understanding and utilizing the latest techniques molecular biology has to offer. In such a manner, we will try to bring this knowledge into the clinics, to try and help our patients as quickly as possible.