Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy
Article Abstract:
Hematopoietic stem cell transplants may be beneficial in treating globoid-cell leukodystrophy. This is a genetic disorder that causes loss of myelin around nerve cells, which results in spasticity, dementia, and peripheral nerve disorders. Researchers gave five children with this disorder a hematopoietic stem cell transplant, which involves transplanting precursors of blood cells. This provides the enzyme that is missing in these patients. Normal enzyme levels were restored in all five children, and neurologic tests and MRI scans revealed that central nervous system degeneration had reversed.
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 1998
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Screening for genetic diseases
Article Abstract:
Many ethical problems must be resolved before groups at risk of genetic diseases be screened. Screening fetuses for some diseases is already done but in many cases, the diseases can be easily treated. Testing is more problematic for diseases for which there is no cure, such as cystic fibrosis. Studies in Montreal and Hong Kong have shown that testing high school students for serious genetic diseases such as Tay-Sachs and thalassemia may effectively reduce the incidence of theses diseases. Teenagers who test positive can decide against having children or make other plans.
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 1997
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