Fetal gene therapy for neurodegenerative disease of infants
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 hepatomegal...
| Main Authors: | , , , , , , |
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| Format: | Journal article |
| Published: |
Springer Nature
2018
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