The Guard1 clinical trial - A first in-human, phase 1/2 study evaluating AVR-RD-02, a hematopoietic stem cell (HSC) gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion

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Bibliographic Details
Published inMolecular genetics and metabolism Vol. 138; no. 2; p. 106991
Main Authors Adera, Mathews, Veselinovic, Milena, Treohan, Anita, Golipour, Azadeh, Ridha, Essra
Format Journal Article
LanguageEnglish
Published Elsevier Inc 01.02.2023
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2022.106991