Magazine article Science News

Marrow for Krabbe's and Twitcher Mice?

Magazine article Science News

Marrow for Krabbe's and Twitcher Mice?

Article excerpt

Marrow for Krabbe's and twitcher mice?

Enzymes occur in the body in very low concentrations and therefore might seem inconsequential, but there are some severe disorders caused by errors in enzyme metabolism. Among these is Krabbe's disease, a rare hereditary condition characterized by a deficiency of galactosylceramidase. Without this enzyme, toxic fat accumulates in the nervous system and in other body tissues. As nerves deteriorate, about four months after birth in most cases, symptoms such as seizures and vision problems appear, followed by death one or two years later. Despite the dire aspects of the disease, however, scientists have two reasons to be optimistic: a good mouse model of the disease and the encouraging results from bone marrow transplant studies, including one reported last week by a group of researchers in the Netherlands, Japan and the United States.

Like a human with Krabbe's disease, the twitcher mouse lacks galactosylceramidase, has brain tissue infiltrated with the abnormal fat-containing globoid cells, and loses the myelin sheaths around nerves. The toxic fat psychosine also accumulates in the central nervous system, and physical symptoms similar to those of Krabbe's disease develop in affected mice, which die about five weeks after birth. In recent years, scientists have used the twitcher mouse to study bone marrow transplants as a way to replace enzyme-deficient cells with normal, enzyme-producing cells. Despite some good results with restoring enzyme activity in the lung and liver, researchers have remained uncertain whether the transplanted cells could cross the selective blood-brain barrier to the tissues most affected by the lack of enzyme. …

Search by... Author
Show... All Results Primary Sources Peer-reviewed

Oops!

An unknown error has occurred. Please click the button below to reload the page. If the problem persists, please try again in a little while.