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Fetal Hemoglobin De-Repression Following CRISPR/Cas9 Mediated Targeting of the gamma-Globin Promoters as a Therapeutic Strategy for gamma-Hemoglobinopathies

Giacomo Frati,Leslie Weber,Tristan Felix,Antonio Casini,Clara Wollenschlaeger,Vasco Meneghini,Mario Amendola,Anna Cereseto,Wassim El-Nemer,Jean-Paul Concordet,Carine Giovannangeli,Marina Cavazzana,Annarita Miccio

MOLECULAR THERAPY（2019）

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