A set of new stem cell lines will make it possible for researchers to explore ten different genetic disorders—including muscular dystrophy, juvenile diabetes, and Parkinson's disease—in a variety of cell and tissue types as they develop in laboratory cultures.
Researchers led by
A set of new stem cell lines will make it possible for researchers to explore ten different genetic disorders—including muscular dystrophy, juvenile diabetes, and Parkinson's disease—in a variety of cell and tissue types as they develop in laboratory cultures.
Researchers led by Howard Hughes Medical Institute investigator George Q. Daley have converted cells from individuals with the diseases into stem cells with the same genetic errors. These newly-created stem cells will allow researchers to reproduce human tissue formation in a Petri dish as it occurs in individuals with any of the ten diseases, a vast improvement over current technology. Like all stem cells, these disease-specific stem cells grow indefinitely, and scientists can coax them into becoming a variety of cell types.
Daley, who is at Children's Hospital Boston, worked with researchers from Harvard Medical School, Massachusetts General Hospital, and the University of Washington to create the disease-specific stem cell strains. The scientists will make the cell lines available to scientists worldwide through a core facility funded by the Harvard Stem Cell Institute. Daley and his colleagues published the details of the disease-specific stem cell lines in an advanced online publication of the journal Cell on August 7, 2008.
"Researchers have long wanted to find a way to move a patient's disease into the test tube, to develop cells that could be cultured into the many tissues relevant to diseases of the blood, the brain and the heart, for example," he says. "Now, we have a way to do just that—to derive pluripotent cells from patients with disease, which means the cells can make any tissue and can grow forever. This enables us to model thousands of conditions using classical cell culture techniques."
Daley's team has created disease-specific stem cell lines for Duchenne muscular dystrophy; Becker muscular dystrophy; juvenile-onset (type I) diabetes; Parkinson's disease; Huntington's disease; Down's syndrome; ADA severe combined immunodeficiency (a form of the disorder commonly known as "boy-in-the-bubble disease"); Shwachman-Bodian-Diamond syndrome (which causes bone marrow failure and a predisposition to leukemia); Gaucher disease (an inherited metabolic disorder in which a fatty substance accumulates in several of the body's organs); and Lesch-Nyhan syndrome (an enzyme deficiency that causes a build-up of uric acid in body fluids). Many more cell lines are possible.
For years, researchers have grown human cells in the laboratory in an attempt to mimic vari