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. 2022 Jun 4;11(6):862. doi: 10.3390/biology11060862
BCL11A BAF Chromatin Remodeling Complex Subunit BCL11A
BDDs blood disease disorders
BMT bone marrow transplantation
Cas CRISPR-associated protein
CRISPR clustered regularly interspaced short palindromic repeat
crRNA sequence-specific CRISPR RNA
EPO erythropoietin
FOG Friend of GATA
FOP Friend of Prmt1
GATA-1 GATA-binding factor 1
GATA-2 GATA-binding factor 2
GVHD graft versus host disease
GETs gene editing tools
HbF hemoglobin F
HDR homology-directed repair
HLA human leukocyte antigen
HPFH hereditary persistence of fetal hemoglobin
HSCs hematopoietic stem cells
IDLVs integrase-defective lentiviruses
iPSCs in pluripotent stem cells
KLF1 Kruppel Like Factor 1
NF-E2 Nuclear Factor, Erythroid 2
NHEJ non-homologous end joining
rAAV6 recombinant adeno-associated viral vectors serotype 6
RBCs red blood cells
SCD sickle cell disease
SCL Stem Cell Leukemia
SOX6 SRY-Box Transcription Factor 6
ssODN single-stranded oligodeoxynucleotide
TALENs transcription activator-like effector nucleases
tracrRNA trans-activating crRNA
ZBTB7A Zinc Finger and BTB Domain Containing 7A
ZFNs zinc-finger nucleases