Extended Data Fig. 1: Characteristics of the patients with FA enrolled in the gene therapy trial. | Nature Medicine

Extended Data Fig. 1: Characteristics of the patients with FA enrolled in the gene therapy trial.

From: Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

Extended Data Fig. 1: Characteristics of the patients with FA enrolled in the gene therapy trial.

a,b, Genotype (a) and hematological and hematopoietic characteristics (b) of the recruited patients. In two patients (FA-02002 and FA-02004), CD34+ cells were collected and then cryopreserved until PB cell counts decreased to levels defined in the gene therapy trial. At that time, cells were thawed, transduced and infused. PB cell counts corresponding to patients FA-02005 and FA-02006 fit the criteria required for both the collection and the infusion of transduced cells. In these two patients, cells were transduced and infused immediately after collection. The table shows PB cell counts and the total content of hematopoietic progenitors per microliter of BM. Additionally, CFC resistance to MMC and percentages of T cells with chromosomal breaks after DEB challenge are shown. CFC values represent mean values corresponding to the scoring of three plates seeded with BM cells obtained before HSC collection and before HSC gene therapy. A total of 25–50 metaphases were analyzed in the DEB tests.

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