AAV |
Adeno-associated viruses |
ABE |
Adenine base editor |
Ad |
Adenoviruses |
AS |
Angelman syndrome |
BE3 |
Base editor 3 |
Cas9 |
CRISPR-associated protein 9 |
CRISPR |
Clustered regularly interspaced short palindromic repeats |
DOGS |
5-Carboxyspermylglycine dioctadecylamide |
DOPE |
1,2-Dioleoyl-sn-glycero-3-phosphoethanolamine |
dsDNA |
Double-stranded DNA |
DSB |
Double-strand break |
E |
Embryonic day |
EGFP |
Enhanced green fluorescent protein |
Eomes
|
Eomesodermin |
EP |
Electroporation |
Fah
|
Fumarylacetoacetate hydrolase |
FACS |
Fluorescence-activated cell sorting |
GFP
|
Green fluorescent protein |
Gria2
|
Glutamate ionotropic receptor AMPA type subunit 2 |
Grin1
|
Glutamate ionotropic receptor NMDA type subunit 1 |
gRNA |
Guide RNA |
HCM |
Hypertrophic cardiomyopathy |
HDR |
Homology directed repair |
HGD |
Hydrodynamics-based gene delivery |
HIV |
Human immunodeficiency virus |
Hpd
|
Hydroxyphenylpyruvate dioxygenase |
HT1 |
Hereditary tyrosinemia type 1 |
Idua
|
α-L-iduronidase |
KD |
Knock-down |
KI |
Knock-in |
KO |
Knockout |
MHCα
|
Myosin heavy chain α |
miRNA |
MicroRNA |
MPS-IH |
Mucopolysaccharidosis type I |
Myh6
|
Myosin heavy chain 6 |
NHEJ |
Non-homologous end joining |
NMDA |
N-methyl-D-aspartate |
NP |
Nanoparticle |
Pcsk9
|
Proprotein convertase subtilisin/kexin type 9 |
PNAs |
Peptide nucleic acids |
PogZ
|
Pogo transposable element derived with ZNF domain
|
RNAi |
RNA interference |
RNP |
Ribonucleoprotein |
rAAVs |
Recombinant adeno-associated viruses |
SaCas9 |
Staphylococcus aureus Cas9 |
Satb2
|
Special AT-rich sequence-binding protein 2 |
Sftpc
|
Surfactant protein C |
ssDNA |
Single-stranded DNA |
Snord115
|
Small nucleolar RNA, C/D box 115 |
snoRNAs |
Small nucleolar RNAs |
Tbr2
|
T-box brain protein 2 |
Tg |
Transgenic |
TPGD |
Transplacental gene delivery |
TPGD-GEF |
Transplacental gene delivery to acquire genome-edited fetuses |
UBE3A
|
Ubiquitin–protein ligase E3A |
UBE3A-ATS
|
UBE3A antisense transcript |
VE |
Visceral endoderm |
YS |
Yolk sac |