March 27 (Bloomberg) -- Jason Burnett and his 10-year-old son Andrew, both born with a genetic defect, have been recruited into an experiment designed to transform bits of their skin into stem cells that may someday hold the key to a cure.
The Burnetts inherited a heart disease that leaves the father exhausted after a short walk. The two are among the first patients working with scientists who are using a new stem-cell technique that may someday revolutionize care for disorders as diverse as diabetes, Alzheimer’s and muscular dystrophy.
Discovered by the Japanese scientist Shinya Yamanaka, the method creates stem cells without using and destroying human embryos. By studying cells created from people with inherited disorders, scientists are observing, in ways never before possible, how diseases progress and react to treatments, said Doug Melton, a Harvard University researcher.
“This is the breakthrough the stem-cell field has been waiting for,” said Beth Seidenberg, a partner at Kleiner Perkins Caufield & Byers, the Menlo Park, California-based venture-capital firm that helped start Google Inc.
Unlike embryonic cells, the cells created using the Yamanaka method opened a path to test drugs for genetic diseases, Seidenberg said.
The approach used in the Burnett family study, being conducted by researchers at the Gladstone Institute in San Francisco, also is being adopted by labs in the U.S., Europe and Asia and helped persuade GlaxoSmithKline Plc to invest $25 million in a joint venture with the Harvard Stem Cell Institute.
Since human embryonic stem cells were first isolated in a laboratory in 1998, they have fired the imagination of doctors, scientists and patients, who envision a day when new tissues or body parts might be grown to replace diseased ones. The cells are pluripotent, meaning they can be turned into any other type of cell, such as those that make up skin, nerves or neurons.
