Fetal gene therapy for neurodegenerative disease of infants
Abstract
For inherited genetic diseases, fetal gene therapy offers the potential of prophylaxis against early, irreversible and lethal pathological change. To explore this, we studied neuronopathic Gaucher disease (nGD), caused by mutations in GBA. In adult patients, the milder form presents with hepatomegaly, splenomegaly and occasional lung and bone disease; this is managed, symptomatically, by enzyme replacement therapy. The acute childhood lethal...
Paper Details
Title
Fetal gene therapy for neurodegenerative disease of infants
Published Date
Jul 16, 2018
Journal
Volume
24
Issue
9
Pages
1317 - 1323
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