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Table 1 Applications of genome editing systems in ALL

From: Targeted genome editing in acute lymphoblastic leukemia: a review

  Outcome Target Gene SSN Technique Modification Type Cell type Reference
Targeting Transcriptional factors Point mutation (insertion/deletion) TAL1 CRISPR-Cas9 HR PEER (in vitro) [4]
Repress expression PAX5 CRISPR-a NA Patient-derived pre-B ALL cells (in vitro) [3]
Knock out NR3C1 CRISPR-Cas9 NHEJ
Knock out LMO2 CRISPR-Cas9 NHEJ PF-382 (in vitro) [5]
Targeting gene fusion expression Chromosomal rearrangement MLL/AF4 TALEN NHEJ K562, HSPCS (in vitro) [42]
Knock in MLL/AF4 AF4/MLL CRISPR-Cas9 HR HEK293 (in vitro) [46]
Chromosomal rearrangement MLL/ENL CRISPR-Cas9 NHEJ HSPCS (in vitro / in vivo, xenograft) [6]
Chromosomal rearrangement MLL/AF9 TALEN NHEJ CD34+ human cord blood (in vivo, xenograft) [43]
Knock in ETV6/RUNX1 CRISPR-Cas9 HR MIFF3 hIPSCs (in vitro) [40]
Drug targts discovery and therapy Knock out CB1 CRISPR-Cas9 NHEJ Jurkat (in vitro) [7]
Knock out BTK CRISPR-Cas9 NHEJ RCH-ACV (in vitro / in vivo, xenograft) [8]
Knock in XPO1 CRISPR-Cas9 HR HL-60, Jurkat, K-562, and MOLT-4 (in vitro / in vivo, xenograft) [57]
Knock out ABCB1 CRISPR-Cas9 NHEJ HALO1 (in vitro) [59]
Modification of CARs Knock out CD19 CRISPR-Cas9 NHEJ NALM6, 697 (in vitro / in vivo, xenograft) [65]
Knock out CD19 CRISPR-Cas9 NHEJ Murine leukemia cell lines E2a:PBX (in vitro / in vivo, xenograft) [83]
Knock out TRAC CRISPR-Cas9 HR NALM6 (in vitro / in vivo, xenograft) [63]
Knock in CD19
Knock out CD52 TALEN NHEJ Two infants (in vivo) [9]
TCR ab
Knock out TCR b CRISPR-Cas9 NHEJ PBMC (in vitro) [84]
Knock out CD7 CRISPR-Cas9 NHEJ T cell lines (in vitro / in vivo, xenograft) [85]
Evolution of pathogenesis Knock out CASD1 CRISPR-Cas9 NHEJ HAP1 (in vitro) [70]
Knock out RIP1 CRISPR-Cas9 NHEJ Patient-derived ALL cells (in vitro / in vivo, xenograft) [75]
Knock out CXCR4 CRISPR-Cas9 NHEJ NALM6 (in vitro / in vivo, xenograft) [10]
Others Knock out FLT3 TALEN NHEJ K562 (in vitro) [86]
Knock out screen NA CRISPR-Cas9 NHEJ NALM6 (in vitro) [87]
Knock out screen NA CRISPR-Cas9 NHEJ MV4,11 (in vitro) [88]
Knock out NUDT15 CRISPR-Cas9 NHEJ Mouse (in vivo) [89]
Knock out DCK CRISPR-Cas9 NHEJ KOPN41 (in vitro) [90]
Knock out PTCH1 CRISPR-Cas9 NHEJ Zebrafish embryos (in vivo, xenograft) [91]
  1. This table shows the main genetic editing studies carried out in ALL, classified according to the target. The different columns indicate: the outcome of edition, the target of edition (highlighted in bold), the technique used, the type of modification, the cell type and the reference