FA |
Fanconi anemia |
CRISPR/Cas9 |
clustered regularly interspaced short palindromic repeats/Cas9 |
BMF |
bone marrow failure |
ZFN |
zinc-finger nucleases |
TALEN |
transcription activator-like effector nucleases |
PARP |
poly(ADP-ribose) polymerase |
PARPi |
PARP inhibition |
gRNA |
guide RNA |
NHEJ |
non-homologous endjoining |
ODN |
oligonucleotide donor |
SNP |
single-nucleotide polymorphisms |
HDR |
Homology directed repair |
MMC |
mitomycin-C |
TERT |
telomerase reverse transcriptase |
HSPC |
hematopoietic stem and progenitor cells |
iPSC |
Induced pluripotent stem cells |