Washington, August 8 : Scientists have achieved a significant breakthrough in creating in the lab a set of new stem cell lines that can make it possible to explore 10 different genetic disorders, including muscular dystrophy, juvenile diabetes, and Parkinson's disease.
Lead researcher George Q. Daley, an expert associated with Howard Hughes Medical Institute, has revealed that the research team converted cells from individuals with the diseases into stem cells with the same genetic errors.
The researcher feels that such stem cells may allow scientists 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.
The disease-specific stem cells grow indefinitely just like all stem cells, and can be coaxed into becoming a variety of cell types.
Daley worked with collaborators from Harvard Medical School, Massachusetts General Hospital, and the University of Washington to create the disease-specific stem cell strains.
A research article in the online edition of the journal Cell says that these cell lines will be made available to scientists worldwide through a core facility funded by the Harvard Stem Cell Institute.
"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," Daley 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," he adds.
So far, the researchers have 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, Shwachman-Bodian-Diamond syndrome, Gaucher disease, and Lesch-Nyhan syndrome.
Daley feels that many more cell lines are possible.
To produce stem cells harbouring the genetic diseases of the donors, Daley and his colleagues mixed cells from patients with the ten disorders with benign viruses to introduce the reprogramming factors into the cells.
Daley says that in many cases these new stem-cell cultures will mimic human disease more reliably than animal models.
According to him, the most immediate application of such stem cells will be to reproduce human diseases in culture to explore their development in different tissues, which may even enable researchers to compare how the same disease varies among people.
The cells will also offer a proving ground for screening drugs to treat disease, Daley says.
He has also revealed that the Harvard Stem Cell Institute will make the stem cell lines available to the scientific community as quickly as possible, and that the institute will also continue to work to generate cell lines for other diseases.
Daley says that his team's techniques for reprogramming adult cells are readily available so other researchers can generate their own disease-specific stem cell lines.
He is planning to collaborate with researchers at other institutions to help produce stem-cell lines for the diseases they want to study.