Gene Therapy, Now in GD1 Trial, Shows Efficacy in Mice at Varying Disease Stages

Gene Therapy, Now in GD1 Trial, Shows Efficacy in Mice at Varying Disease Stages

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Gene Therapy, Now in GD1 Trial, Shows Efficacy in Mice at Varying Disease Stages

An investigational gene therapy aiming to restore GBA gene function — which is defective in people with Gaucher disease — showed efficacy both before and after symptom development in a mouse model of the disorder, a study reported.  These findings supported the ongoing GuardOne Phase1/2 clinical trial, which is evaluating the potential gene therapy AVR-RD-02 as a treatment of type 1 Gaucher disease (GD1). The study, “Correction of pathology in mice displaying Gaucher disease type 1…

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