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Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy (2025)

First Author: Geard A

Attributed to: MICA: Towards clinical trial readiness of gene therapy for Niemann-Pick disease type c funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.3390/pharmaceutics17050650

Publication URI: http://dx.doi.org/10.3390/pharmaceutics17050650

Type: Journal Article/Review

Parent Publication: Pharmaceutics

Issue: 5