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CLINICAL RESEARCH: Gene Therapy a Suspect in Leukemia-like Disease

✍ Scribed by Marshall, E.


Book ID
121180427
Publisher
American Association for the Advancement of Science
Year
2002
Tongue
English
Weight
462 KB
Volume
298
Category
Article
ISSN
0036-8075

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## Abstract Pompe disease is a rare, progressive disease leading to skeletal muscle weakness due to deficiency of the acid α‐glucosidase (GAA) enzyme. Herein we report the first diagnosed Finnish patient with a phenotype compatible with the late‐onset form of Pompe disease. Molecular genetic analys