I remember watching the Jerry Lewis Labor Day Telethon as a child, wondering why there was no cure for muscular dystrophy, and hoping that someone would discover the key to success. Little did I suspect that years later my own career would be focused on finding a cure for children with MD. This past year brought a new perspective to my view of childhood diseases, as my wife, Joel, and I had our first child. Sitting with my daughter, Jacqueline, watching this year's Telethon led me to think about how I came to work on muscular dystrophy and what still needs to be done to find that elusive cure for the thousands of children who watch Jerry every year. I thought I would share some of those thoughts and provide some insight into where our research efforts stand.

Aside from those Labor Day hours in front of the television set, muscle diseases rarely came to my mind while growing up. After graduating from Rice University in Houston, I wanted to study how the human body develops from a single egg into a complex network of organs.

I enrolled in graduate school at the University of Washington in Seattle to study molecular biology and soon found myself working with Stephen Hauschka, who had perfected methods to grow muscle cells in the laboratory. These cells provided a powerful system to understand the complexities of human development, using muscle as a model. We were studying how muscle genes could be isolated and how those genes controlled the fate of individual muscle cells.

This was considered basic research in its purest form, so it was with amazement I found that much of our work was supported by the Muscular Dystrophy Association. Why would MDA invest in a young student to research how muscle tissue forms in the absence of a disorder? As I later realized, this type of research, supported by MDA for years, led directly to many of the techniques, resources and personnel that are now closing in on treatments for numerous neuromuscular diseases.

In 1980, little was known about genes and how they contributed to muscular dystrophy. By taking a focused, long-range view, MDA made it possible to seriously embark upon finding a treatment for what physicians had long considered to be incurable diseases.

After completing my doctoral degree, I decided to continue my work by directly studying the gene that causes Duchenne muscular dystrophy (DMD). Because of enormous leaps of knowledge in the 1980s, it became possible to seek directly the genes that cause genetic diseases.

Within a week of my graduation I heard from researchers Louis Kunkel at Children's Hospital of Boston and Ron Worton at the Hospital for Sick Children in Toronto that the gene for DMD was likely in hand. For the first time we were about to learn the fundamental cause of this disease and might even be able to contemplate how it could be cured.

With this realization, I moved back to Houston to work with C. Thomas Caskey on a newly discovered mouse that developed DMD. Caskey reasoned correctly that this mouse model would allow us to study DMD in ways that could never previously have been approached. Over the next few years we identified the mouse DMD gene and began a series of experiments that proved, for the first time, that muscular dystrophy could be cured. It's no great surprise that this research was paid for by MDA.

In 1990 I moved to Ann Arbor, Mich., and established my own research team at the University of Michigan. My goal was simple: Find a cure for muscular dystrophy. Seven years later, with a team of 15 students and young scientists, we have identified how a cure might be developed and have set in motion a huge effort aimed at achieving that goal.

Over these years I have spent considerable time at MDA-sponsored conferences, meeting other scientists, discussing strategies and working with labs throughout the world to help find ways to conquer this disease. Many people have commented to me that if we were able to put a man on the moon, then why haven't we yet cured muscular dystrophy? The answer to that question is sadly simple: Putting a man on the moon was easy compared with the obstacles we face in treating a disease whose origin lies in our own genetic material. I remind myself that not too many years ago few believed we would walk on the moon, but we did. Today we know muscular dystrophy can be cured; it's only a question of when.

What is being done to make this cure a reality? The simple answer is that a wide array of strategies are being pursued, any of which could lead to a huge breakthrough. Drug treatments, genetic counseling, myoblast transfer and gene therapy all show promise in various forms. It's likely that each of these approaches will be of importance in treating some of the 40 MDA-supported diseases. Many laboratories are still performing the nuts and bolts basic science needed to make the next breakthrough possible, while others are focused on harnessing what is already known and bringing it into the clinic. My lab sits somewhere in between, studying the mouse to develop a cure for use in the clinic.

Our approach is through gene therapy: to use genes as medicine by delivering a normal version of the disease-causing gene to dystrophic muscle cells. My lab has shown that the dystrophin gene, which is the largest known gene, can be modified in numerous ways so that it can be inserted into biological shuttles, or "vectors," that deliver genes to muscle. We have also found a new way to make the delivery shuttles. By taking a cold virus known as adenovirus and removing all the viral genes, we've been able to pack a normal dystrophin gene into the virus. Removing the viral genes was critical, as these can trigger rejection of the treated muscle by a person's natural immunity. Finally, we have a vector with the potential to deliver a miniature factory capable of producing normal dystrophin, but one which should not lead to self-destruction of the treated muscle.

Even with these new tools in hand, there are several remaining concerns that must be addressed. A number of minor changes will almost certainly be needed to increase the efficiency of the vectors and enable large-scale production and purification of them.

In what may be the greatest challenge, we must find ways to get these viruses to muscles throughout the human body.

Finally, we must also show that these new viral vectors can be used safely, without toxicity or side effects. This latter challenge will require small-scale clinical trials even before one can hope to test whether the viruses are effective in fighting muscular dystrophy. It's inevitable that problems will be found, requiring additional modifications back in the laboratory to create an approach that really works in people, and not just on paper.

These approaches are being pursued jointly by many laboratories, ensuring a wide source of fresh ideas. Of great importance in this endeavor, MDA continues its support of young scientists in the hope that they will grow into productive researchers who may find that final, missing key. A number of challenges remain, and I suspect I'll be watching a few more Telethons before DMD is cured -- but, I hope, not many.

Jeffrey Chamberlain is a long-time MDA grantee whose work at the University of Michigan in Ann Arbor has been crucial in the development of gene therapy strategies for Duchenne muscular dystrophy.