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Thus, CRISPR/Cas9-based gene therapy has reached the clinical trial stage for many monogenic diseases such as sickle cell anemia [7], -thalassemia [7], hereditary tyrosinemia type I [8] and is being applied in advanced preclinical testing stages of Duchenne muscular dystrophy (DMD), hemoglobinopathies, and hereditary tyrosinemia type I

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[DOI] [PubMed] [Google Scholar] 251.Nakayama E, Akiba T, Marumo F, Sato C