Stem Cells Created To Treat Genetic Disorders

Scientists said that they have created stem cells based on ordinary skin Stem Cells Created To Treat Genetic Disorders and bone marrow cells for 10 genetic disorders, which will enable them to study the development of the disease in a lab dish.

The teams from Harvard Medical School, Children’s Hospital in Boston, Massachusetts General Hospital, and the University of Washington, said a variety of diseases such as Parkinson’s, Huntington’s and Down syndrome could be researched and watched in a lab, as the disease progressed and could help researchers finding treatments.

Reported online in the journal Cell, the researchers said they reprogrammed skin cells from two elderly patients with ALS, or Lou Gehrig's disease, and grew them into nerve cells.

The researchers said that this could speed medical research and they plan on sharing the discovery with other researchers and fellow scientists.

Dr. George Daley and his colleagues at the Harvard Stem Cell Institute said in the new technique the cells are reprogrammed giving them qualities that enable them to transform into all types of tissue, be it the heart, nerve or brain.

According to Doug Melton, co-director of the institute, this new method would help researchers "watch the disease progress in a dish, that is, to watch what goes right or wrong, I think we'll see in years ahead that this opens the door to a new way to treating degenerative diseases," he said.

Called iPS cells, the new cells are made by a technique discovered by Shinya Yamanaka of Kyoto University in Japan. This reprograms cells, giving them the qualities of embryonic stem cells and similar to the ordinary stem cells, these new induced pluripotent stem cells can be cultured into any desired tissue, be it from the heart muscle cells, blood cells or brain cells. This new method gives an alternative to the embryonic technique which raised controversies as it involves using human eggs.

Melton said a new laboratory to serve as a repository for the cells has been made, and 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."

"The hope is that this will accelerate research and it will create a climate of openness," said Daley. He felt this was just the beginning and this stem cell research could be used for many other diseases. "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."

The work was funded by the National Institutes of Health as well as private contributions to the Harvard Stem Cell Institute.