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 | |||
TXNIP | ||||||
CB2 | ||||||
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] |
MLL/AF9 | ||||||
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] | |
AF9/MLL | ||||||
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] |
CB2 | ||||||
Knock out | BTK | CRISPR-Cas9 | NHEJ | RCH-ACV (in vitro / in vivo, xenograft) | [8] | |
BLK | ||||||
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] | |
PAX5 | ||||||
EBF1 | ||||||
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] | |
TRAC | ||||||
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] |