Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy
<b>Background/Objectives</b>: Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the <i>GBA1</i> gene. Type 1 Gaucher disease is characterised by substrate accumulation in the visceral organs, which occurs in combination with acut...
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| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2025-05-01
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| Series: | Pharmaceutics |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1999-4923/17/5/650 |
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