Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy

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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Bibliographic Details
Main Authors: Amy F. Geard, Giulia Massaro, Michael P. Hughes, Patrick Arbuthnot, Simon N. Waddington, Ahad A. Rahim
Format: Article
Language:English
Published: MDPI AG 2025-05-01
Series:Pharmaceutics
Subjects:
Gaucher disease
AAV
gene therapy
mouse model
GD Type 1
Online Access:https://www.mdpi.com/1999-4923/17/5/650
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