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Major Therapy Category
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DNA therapy
Genetically-modified cell therapy
RNA therapy
Therapy Type
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Aptamer
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DNA vaccine
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miRNA
mRNA
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AG
AIPL1
Apollon
AR
ATP6V1A
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BCMA
C1s
CD19
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CDK4
CDK5
CDK6
CHRNA5
circPVT1
Claudin-7
cMet
CMTR1
COP1
COX-2
CRX
CSN5
CT16
DDX3X
DGAT2
DMD
Dnm3os
DNMT1
E2F-1
EDA
ELF5
EpCAM
ETS2
ETV6
EZH2
FAH
FANCA
FEN1
FGFR4
FIX
FLAP
FOXD1
FOXM1
GAA
GATA6
GD2
Girdin
Gp100
GRAMD1B
GRHL2
GSE1
HA
HAS2
HDAC4
HDAC6
hemagglutinin
HIV-1gag
HIV-1nef
HIV-1pol
HOXB7
HPV-E6
HTT
IFNα
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IGF-I
IGF2BP1
IL-2
IL-27
IL12a
IL12b
ING2
IQGAP3
JAK1
JP2
Kcna1
KRT14
lacZ
LAMA3
LAPTM4B
LINC00461
LINC00511
LINE-1
LKB1
luciferase gene
Mapk1
MAPT
mCD19
MCM3
Mecp2
MEM49
MFN2
miR-155
miR-211
miR-22
miR-26a
miR-28
miR-29a
miR-29b
miR-302b
miR-34a
MOG
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MYBL2
MYC
NCL
NeuroD1
NRAS
NRCP
NRF1
NUP93
NY-ESO-1
OCT4
PABPN1
PARP1
PARP2
PDL1
Pln
PMP22
POGLUT1
Pparg
PRMT1
PTHrP
RS1
S
S100PBP
Set8
SHH
SIM2
SIVenv
SIVgag
SIVpol
SMARCA4
SMN2
SNHG15
SOD1
SRC-1
SRF
SRSF7
STAT6
TAK1
TCF7L2
Tert
TNFR1
TRAP1
Trp2
Trp53
UHRF1
Upf1
Upf3b
Utx
Xkr8
YAP/TAZ
ZEB1
ZMPSTE24
Treatment
All Treatment
AAV-CRISPR/Cas12a-del_46–54
AAV-CRISPR/Cas9-del_46–54
AAV-FIX
AAV-GAA
AAV-mediated CRX
AAV-miR SOD1
AAV-NeuroD1
AAV-optPABPN1
AAV-PHP.eB-iMecp2
AAV-shRNA3x
AAV2/8-LSPhGAApA53
AAV2/9-JP2NT
AAV8-miR-22
AAV8-scRS/IRBPhRS
AAV9-AIPL1co
AAV9-AIPL1wt
AAV9-CRISPRa-sgKcna1
AAV9-HDAC4
AAV9-Tert
AAV9.Eno2-MFN2*R94Q
Ad-E2F-1
Ad-LacZ
Ad.Shh
AdHA-PPARg2
AdHA-PPARgCDE
AG-specific aptamers
Apollon siRNA
ARCT-021(ARCT COVID-19 vaccine)
AS1411 aptamer
ASO-001933
ATP6V1A siRNA
BCMA CAR-T cell
BNT162b mRNA vaccine
BNT162b2 mRNA vaccine
C1s siRNA
CD19 CAR-T cells
CD24 CAR-T cells
CDA siRNA
CDK5 siRNA
Ce-49 sh aptaDiR
CHRNA5 siRNA
Claudin-7 siRNA
cMet CAR-T cells
CMTR1 siRNA
Cobomarsen
COP1 siRNA
COX-2 siRNA
CRISPR-Cas9 nickase
CRISPR/Cas9-KRT14
CSN5 siRNA
CT16 siRNA
DDX3X siRNA
DGAT2-GalNac 1473 siRNA
EDA CAR-T cells
ELF5 siRNA
ERK-dimerization inhibitory peptide virus
ETS2 siRNA
ETV6 siRNA
exoASO-STAT6
EZH2 siRNA
FEN1 siRNA
FGFR4 siRNA
FLAP siRNA
FoxM1 siRNA
GD2 CAR-T cells
ghR-34a
GRAMD1B siRNA
GSE1 siRNA
HD-RAd-IGF-I
HOXB7 siRNA
HPV-E6 and Set8 siRNAs
HPV-E6 siRNA
IL-27 plasmid DNA
influenza H5N1 DNA vaccination
ING2 siRNA
L17-F05 mRNA vaccine
LAPTM4B siRNA
lentiviral CRISPR epigenome editing system
LINC00511 siRNA
LINE-1 ASO
LKB1 siRNA
LMP sh.Trp53.393
LPC-ATP7B-Heps
LV-CMV-LAMA3
LV-FAH
LV-FANCA Autologous CD34 + Cells
LV-IFNα
LV-IFNγ
LV-Mpl
MEM49-encoding DNA vaccine
Merck Ad5/HIV vaccine
miR-211
miR-26a mimic
miR-28 mimics
miR-302b mimic
miR-34a mimics
MIRTX (completely opposite complementary strand to miRNA-29b-1-5p)
miR‐155 oligonucleotide inhibitor
mix siRNA CDK4/6 (containing CDK4 siRNA and CDK6 siRNA)
MOG DNA vaccine
mouse (m)IL12-mRNA construct
MRG-106
mRNA vaccine
mRNA-based universal vaccine
MYC siRNA1
MYC siRNA2
Neocarzinostatin-EpCAM Aptamer
NRAS siRNA-1
NRAS siRNA-2
nusinersen
NY-ESO-1 TCR-T
PDL1 siRNA
pDnm3os
pIL-2
Pip6e-PMO
PLN ASO
PMP22 ASO1
pRhPD-1-p27 DNA vaccine
PRMT1 siRNA
PTHrP siRNA
pVax-luc
pVax-PSA
S100PBP siRNA
sCA-miR-29a-3p
sCA-miR-29b-3p
shEZH2
si ZEB1
si-circPVT1-1
si-circPVT1-2
si-Girdin
si3033
siAR
siCAG/CUG trinucleotide repeat (TNR)
siCGA/UCG trinucleotide repeat (TNR)
siFOXD1-1
siFOXD1-2
siFOXM1
siGATA6
siGRHL2
siHAS2
siHDAC6
siIGF2BP1
siIQGAP3
siLINC00461
SIM2 siRNA
siMCM3
siMYBL2
siNRCP
siNRF1
siNUP93
siOCT4
siPARP1
siPARP2
siSNHG15
siSRF
siTRAP1
siUHRF1
SIV DNA vaccine
siZMPSTE24
SMARCA4 siRNA
Spinraza®
SRC-1 siRNA
SRSF7 siRNA
SW0123 mRNA vaccine
T208 lncRNA antisense oligo
TAK1 siRNA
TCF7L2 siRNA
Upf1 ASO
Upf3b ASO
Utx shRNA
Xkr8 siRNA
YAP/TAZ siRNA
Organism
All Organism
Canis lupus
Homo sapiens
Macaca mulatta
Mus musculus
Rattus norvegicus
Sus scrofa
Strategy
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Microarray
RNA-seq
scRNA-seq
Ex vivo/In vivo
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ex vivo
in vivo
Disease Group
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Autoimmune Disease
Cancer
Genetic Disease
Infection
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