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Base Editing of Gamma Globin Gene Promoters Generates Durable Expression of Fetal Hemoglobin for the Treatment of Sickle Cell Disease

Adrian P. Rybak, Elsie Zahr Akrawi,Conrad Rinaldi,Scott J. Haskett,Ling Lin,Jeffrey Marshall,Alexander Liquori,Luis Barrera,Jenny Olins,S. Haihua Chu,Jeremy Decker,Minerva Sanchez,Yeh-Chuin Poh, Matt Humes,Michael S. Packer,Nicole M. Gaudelli,Sarah Smith,Adam Hartigan,Giuseppe Ciaramella

MOLECULAR THERAPY（2020）

Cited 2|Views19
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