Drug target identification and validation

Ready-to-go knockout cell models 

The ready-to-go knockout range has over 7,500 products available. Find the relevant cell line for your research easily by searching for your gene of interest.

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Examples of popular knockout cell lines

ABAT

ABCA1

ABCC1

ACE

ACHE

ACVR2A

ADA

ADIPOR1

ADIPOR2

ADK

ADORA2A

ADORA2B

ADRA1A

ALAD

ANXA1

AR

ASIC1

ATP2C1

BCHE

BLVRB

CA2

CACNA1H

CACNA2D1

CACNA2D2

CACNB1

CACNB2

CACNB3

CACNB4

CCKBR

CCND1

CHD1

CHRNB2

CKB

CLCN2

CNR1

COMT

CPT1A

CPT2

CYP51A1

DPP4

EPOR

ESRRG

FADS1

FASN

FGF2

FN1

FTL

GAA

GABBR1

GABRA5

GABRB3

GANAB

GANC

GGCX

GLRB

GSR

GSS

HMMR

HPRT1

HTR6

IFNAR1

IFNAR2

IFNGR1

IFNGR2

IL11RA

IMPDH1

ITGA2B

JUN

KCNC1

KCND2

KCNH2

KCNJ11

KCNK2

KCNQ1

KCNQ2

KEAP1

KMT2C

M6PR

MAOA

MAP1A

MAP2

MAP4

MME

MMP11

MMP15

MMP16

MMP17

MMP24

MMP25

MS4A2

MTR

MUT

NOS2

NR3C2

PDE10A

PDE4A

PDE5A

PDE7A

PDE8A

PDXK

PGF

PLAU

PLAUR

PNP

POLE4

PRKAG1

PRKAG2

PRLR

PROS1

PSMB10

PSMB8

PSMB9

PTGER3

QPRT

RAMP1

RAMP2

RARG

RFK

RXRA

RXRB

S1PR5

SCN1B

SCN5A

SCNN1D

SHMT1

SIGMAR1

SLC18A2

SLC5A2

SMS

SOAT1

SQLE

SRD5A1

SYT2

THRB

TOP1MT

TOP2B

TPMT

TSPO

TUBA1A

TUBA4A

TUBB1

TUBB4B

TUBG1

UGCG

VAMP1

VEGFA

VEGFB

VKORC1

 

 

Drug target identification and validation

Biological target identification and validation are among the most important steps in developing a new drug. Drug target validation involves proving that either DNA, RNA, or a protein molecule is directly involved in a disease process and can be a suitable target for development of a new therapeutic drug. 

To qualify as ‘druggable’, a target must be accessible to the proposed drug molecule, and a measurable biological reaction must be provoked as a consequence of the drug interacting with the target. This reaction may be measured both in vitro and in vivo

Once the target has been identified, for example by genomic or proteomic investigations, the target is then validated in functional studies. The main strategies involved in target validation are gene knockout studies and direct inhibition of the target by small molecules, peptides, antibodies or any other class of inhibitors. The knockout approach genetically inhibits the target, thereby mimicking the action of the drug and allows a drug to be modeled before it is developed.

Information about established drugs and drug-target interactions is available online via public databases such as Drug Bank and the Therapeutic Targets Database. In addition, information on potentially novel drug targets can be found at Center Watch's, Drugs in Clinical Trials Database, which identifies trends in drug development based on current clinical trials.