Stem cells are cells found in most, if not all, multi-cellular organisms. They are characterized by the ability to renew themselves through mitotic cell division and differentiating into a diverse range of specialized cell types. Harvard scientists say they have created stems cells for 10 genetic disorders, which will allow researchers to watch the diseases develop in a lab dish.
This early step, using a new technique, could help speed up efforts to find treatments for some of the most confounding ailments, the scientists said. The new work was reported online Thursday in the journal Cell, and the researchers said they plan to make the cell lines readily available to other scientists.
Dr. George Daley and his colleagues at the Harvard Stem Cell Institute used ordinary skin cells and bone marrow from people with a variety of diseases, including Parkinson’s, Huntington’s and Down syndrome to produce the stem cells.
The new cells will allow researchers to “watch the disease progress in a dish, that is, to watch what goes right or wrong,” Doug Melton, co-director of the institute, said during a teleconference.
The new technique reprograms cells, giving them the chameleon-like qualities of embryonic stem cells, which can morph into all kinds of tissue, such as heart, nerve and brain. As with embryonic stem cells, the hope is to speed medical research.
Melton said the new disease-specific cell lines “represent a collection of degenerative diseases for which there are no good treatments and, more importantly, no good animal models for the most part in studying them.”
A new laboratory has been created to serve as a repository for the cells, and to distribute them to other scientists researching the diseases, Melton said. “The hope is that this will accelerate research and it will create a climate of openness,” said Daley.
He expects stem cell lines to be developed for many more diseases, noting, “this is just the first wave of diseases.” Other diseases for which they created stem cells are Type 1, or juvenile, diabetes; two types of muscular dystrophy, Gaucher disease and a rare genetic disorder known as the “bubble boy disease.”
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