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

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Title: Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy.
Authors: Geard, Amy F.1,2 (AUTHOR), Massaro, Giulia1,2 (AUTHOR), Hughes, Michael P.1,3 (AUTHOR), Arbuthnot, Patrick1,2 (AUTHOR), Waddington, Simon N.2,3 (AUTHOR), Rahim, Ahad A.1,3 (AUTHOR) a.rahim@ucl.ac.uk
Source: Pharmaceutics. May2025, Vol. 17 Issue 5, p650. 22p.
Database: Academic Search Ultimate
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ISSN:19994923
DOI:10.3390/pharmaceutics17050650