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A Novel Oligonucleotide-Based RNA Base Editing Therapeutic Approach for the Treatment of Hurler Syndrome
May 2020, 23rd ASGCT Annual Meeting, Oral Presentation
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Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34+ Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major
December 2019, 61st ASH Annual Meeting, Poster Presentation
