The National Institutes of Neurological Disorders and Stroke recently awarded the Tulane Center for Gene Therapy $1,039,500 to research the therapeutic use of adult stem cells from bone marrow to treat lysosomal storage diseases, inborn errors of metabolism that cause central nervous system defects.

"Lysosomes are the cellýs recycling centers," said the lead investigator, Donald G. Phinney. "When lysosomes can not do their job, an individual may experience a variety of symptoms including organ dysfunction, skeletal abnormalities and neurological disorders, which lead to a shortened life expectancy."

Phinney also recently co-authored an article in "Stem Cells" that describes the first comprehensive analysis of adult stem cells at the molecule level, and in part explains their ability to generate many different types of cells. It is this ability that makes stem cells useful in therapies for human diseases, such as lysosomal storage diseases. There are over 20 types of lysosomal storage diseases described in humans. Over half of these diseases cause progressive mental deterioration for which there is no treatment currently available.