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. 2024 Mar 22;9(6):e171844. doi: 10.1172/jci.insight.171844

Figure 1. Genome engineering of GNTI-122 from CD4+ T cells.

Figure 1

(A) Resulting genome-engineered product of GNTI-122. Digital PCR results of transgene integration at the (B) FOXP3 and (C) TRAC loci of mock-engineered (Mock) and GNTI-122 cells from healthy donors (HDs) and patients with type 1 diabetes (T1D). (D) Representative FACS analysis of GNTI-122 and Mock cells generated from a patient with T1D, before enrichment to after enrichment with rapamycin (n = 3). Cells were stained 3 days after editing before enrichment to determine the initial TCRVb13.6+FOXP3+ editing frequency and at the time of cryopreservation after enrichment to determine purity. (E and F) Graphical representation of 3 independent batches of GNTI-122 cells and their corresponding Mock cells generated in parallel, from 3 HDs and 3 donors with T1D. Graphs represent the TCR single-positive population and dual-engineered populations. FRB, FKBP-rapamycin binding domain; MND, myeloproliferative sarcoma virus enhancer, negative control region deleted, dl587rev primer-binding site substituted; CISC, chemically inducible signaling complex.