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Wen PY, Schiff D, Cloughesy TF, Raizer JJ, Laterra J, Smitt M et al

They imagine the protocol working silently in the background, quietly correcting years of fatigue, low drive, and sluggish body composition while their daily habits stay exactly the same

Since the mVCN of the cell product that was infused into these two patients was 1.9 and 2.1, the relatively low mVCN determined 24 months later in the peripheral blood of these two patients indicate that the modified cells may have failed to properly engraft, survive and/or produce -globin (and in consequence hemoglobin), and were therefore unable to meaningfully contribute to hematopoiesis and support correction of the disease

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According to the expression of concern published on 8 May, the Editorial Board has decided to allow the authors to confirm the validity of the overall findings by performing re-experimentation on certain elements of this study. Lu did not respond to an emailed request for a comment about why the two studies were treated differently

See ophthalmologist immediately ruling out treatable progression