CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and B-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable.
Michelle I. Lin,Elizabeth Paik,Bibhu Mishra,David Burkhardt,Andrew Kernytsky,Michael Pettiglio, Yi-Shan Chen,Kaleigh Tomkinson,Amanda Woo,Mauricio Cortes, Siyuan Tan, Todd D. Borland, Lawrence Klein, Angela Yen,Sudipta Mahajan, Eric Chan,Brenda Eustace,Matthew Porteus,Tirtha Chakraborty,Chad Cowan,Rodger Novak,Ante Lundberg Blood（2017）
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