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A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.

Cai L, Bai H, Mahairaki V, Gao Y, He C, Wen Y, Jin YC, Wang Y, Pan RL, Qasba A, Ye Z, Cheng L
Stem Cells Transl Med. 2018 Jan;7(1):87-97. doi: 10.1002/sctm.17-0066. Epub 2017 Nov 21. (Link opens in a new window) PubMed (Link opens in a new window) Article

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