CureDuchenne - Together we Can CureDuchenne
June 2013

Scientist of the Month - Thomas Rando, M.D., Ph.D.

Thomas Rando, M.D., Ph.D.
Thomas Rando, M.D., Ph.D.

Thomas Rando, M.D., Ph.D., professor of neurology and neurological sciences at Stanford University School of Medicine and director of Stanford’s Glenn Laboratories for the Biology of Aging, has focused his entire career researching muscular dystrophy.

The main areas of interest of the Rando Laboratory are muscle stem cell biology, muscle stem cell aging, muscular dystrophies, tissue engineering and basic muscle cell biology. Dr. Rando’s research focuses on the restorative and repair mechanism of stem cells. The lab has a long-standing interest in understanding the mechanisms of cell injury and cell death in muscular dystrophies and the development of novel therapeutics. The long term goal is to develop stem cell therapies for Duchenne muscular dystrophy.

The Rando Laboratory has seen success in mouse models. They have obtained muscle biopsies from patients, isolated them and grown them extensively to transplant the cells into mice to test them. Their research on muscle stem cells is turning toward therapeutic applications. A major emphasis of their recent effort is pre-clinical studies of using muscle stem cells in models of cell transplantation to treat muscular dystrophies with an eye toward future human trials.

Dr. Rando is the senior author of a study that was published online recently in the Journal of Clinical Investigation. His lab created a mouse model of muscular dystrophy in which regenerating muscle tissues gives off visible light from luciferase, the protein that causes fireflies’ tails to glow. When the luciferase gene is activated in a cell it glows whenever the mice are given a compound that gives off light in the presence of luciferase. So as the muscular dystrophy progresses the muscle that is being repaired becomes increasingly luminescent. This mouse model paves the way to quicker, cheaper and more accurate assessment of the efficacy of therapeutic drugs. It is already being used to test stem cell and gene therapy approaches for muscular dystrophy. Click here to read the abstract.

“These are reporter mice because they report on a disease progression,” said Dr. Rando. “Using mice allows us to see the progression of the disease in a compressed time scale and be able to test other approaches.”

Research takes time but progress is being made in the Duchenne field.

“We know the time pressure for parents with children with Duchenne is profound and that sense of urgency is shared by scientists,” said Dr. Rando. “I know the slow pace of research can be frustrating. You never know which avenues will lead to treatments and sometimes they come from avenues that are unexpected. That is why it is good to do a combination of both targeted therapeutic research and basic research that could lead to new therapeutic opportunities. We are optimistic that stem cell therapies will be in more and more trials that will make a huge difference in the lives of boys with Duchenne.”

Dr. Rando is a member of the CureDuchenne Scientific Advisory Board.

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