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More Options for Gene Editing in Hemoglobinopathies
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DOI：10.1056/NEJMe2602194.png)
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Abstract
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Transfusion-dependent beta-thalassemia and sickle cell disease, both characterized by defective hemoglobin synthesis, are the most common monogenic diseases worldwide. Each year, approximately 60,000 infants are born with transfusion-dependent beta-thalassemia, and 500,000 infants receive a diagnosis of sickle cell disease.(1,2 )Historically, patients with these disorders have rarely survived beyond the first two or three decades of life, but advances in treatment strategies and therapeutic options, involving the availability of safe blood products and the improvements in conventional therapies, have resulted in an extended lifespan and higher quality of life among patients in high-income countries.(1,2) There remains an urgent need for the . . .
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