Design and outcome measures for a phase 1/2 dose-finding study to evaluate systemic administration of an AAV9-based gene therapy for peripheral manifestations of Gaucher disease
| Autor: | Neuhaus, Sarah, Tamburri, Paul A., Weaver, Michael A., Booth, Jeff, Mahoney, Erin, Shaughnessy, Lee, Sondergaard, Patricia, Hatch, Daniel A., Lewis, Travis B., Uspenskaya, Olga, Sevigny, Jeffrey |
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| Zdroj: | In Molecular Genetics and Metabolism February 2023 138(2) |
| Databáze: | ScienceDirect |
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