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. Author manuscript; available in PMC: 2011 May 11.
Published in final edited form as: N Engl J Med. 2010 Nov 11;363(20):1918–1927. doi: 10.1056/NEJMoa1003548

Figure 3. Sustained Polyclonal Hematopoietic Repopulation after Gene Therapy.

Figure 3

In Panels A and B, retroviral insertion sites (RISs) were detected by linear-amplification–mediated polymerasechain-reaction (LAM-PCR) analysis of transduced hematopoietic cells at various days after gene therapy from bone marrow cells and primary blood leukocytes obtained from Patients 1 and 2. CD3 denotes T cells, G granulocytes, HC healthy control subject, IC internal vector control, and M 100-bp marker. In Panels C and D, the relative contribution of individual clones retrieved from bone marrow cells and primary blood leukocytes is estimated in sequenced polyclonal-cell samples from Patients 1 and 2. The presence of individual gene-corrected cells is given as the percentage of all LAM-PCR amplicon sequence reads identified in a particular sample. The 10 most frequently sequenced RISs are ranked from 1 to 10 according to retrieval frequency. Genes that are highlighted in gray carry a RIS that was detected at more than one time point within the 10 most frequently sequenced sites. Also listed are the numbers of other unique, mappable RISs that were sequenced less frequently than the first 10 in the respective sample analyzed, which are labeled “others.”