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CEDAR Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe Sickle Cell Disease

John F. Di Persio,Julie Kanter,Patrick Leavey,David C. Shyr,Alexis A. Thompson,Matthew H. Porteus,Allison Intondi,Premanjali Lahiri,Daniel Dever,Alexandria Petrusich,Joshua Lehrer-Graiwer

MOLECULAR THERAPY（2022）

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