TARGETID

T83585

FORMERID

TTDI02221

UNIPROID

WASP_HUMAN

TARGNAME

Wiskott-Aldrich syndrome protein (WAS)

GENENAME

WAS

TARGTYPE

Clinical trial target

SYNONYMS

WASp; IMD2

FUNCTION

Important for efficient actin polymerization. Possible regulator of lymphocyte and platelet function. Mediates actin filament reorganization and the formation of actin pedestals upon infection by pathogenic bacteria. In addition to its role in the cytoplasmic cytoskeleton, also promotes actin polymerization in the nucleus, thereby regulating gene transcription and repair of damaged DNA. Promotes homologous recombination (HR) repair in response to DNA damage by promoting nuclear actin polymerization, leading to drive motility of double-strand breaks (DSBs). Effector protein for Rho-type GTPases that regulates actin filament reorganization via its interaction with the Arp2/3 complex.

PDBSTRUC

2OT0; 2K42; 2A3Z; 1T84; 1EJ5

SEQUENCE

MSGGPMGGRPGGRGAPAVQQNIPSTLLQDHENQRLFEMLGRKCLTLATAVVQLYLALPPGAEHWTKEHCGAVCFVKDNPQKSYFIRLYGLQAGRLLWEQELYSQLVYSTPTPFFHTFAGDDCQAGLNFADEDEAQAFRALVQEKIQKRNQRQSGDRRQLPPPPTPANEERRGGLPPLPLHPGGDQGGPPVGPLSLGLATVDIQNPDITSSRYRGLPAPGPSPADKKRSGKKKISKADIGAPSGFKHVSHVGWDPQNGFDVNNLDPDLRSLFSRAGISEAQLTDAETSKLIYDFIEDQGGLEAVRQEMRRQEPLPPPPPPSRGGNQLPRPPIVGGNKGRSGPLPPVPLGIAPPPPTPRGPPPPGRGGPPPPPPPATGRSGPLPPPPPGAGGPPMPPPPPPPPPPPSSGNGPAPPPLPPALVPAGGLAPGGGRGALLDQIRQGIQLNKTPGAPESSALQPPPQSSEGLVGALMHVMQKRSRAIHSSDEGEDQAGDEDEDDEWDD

DRUGINFO

OTL-103

DRUGINFO

Hematopoietic stem cell gene therapy

DRUGINFO

WASP gene therapy

KEGGPATH

hsa04062:Chemokine signaling pathway

KEGGPATH

hsa04520:Adherens junction

KEGGPATH

hsa04666:Fc gamma R-mediated phagocytosis

KEGGPATH

hsa04810:Regulation of actin cytoskeleton

KEGGPATH

hsa05100:Bacterial invasion of epithelial cells

KEGGPATH

hsa05130:Pathogenic Escherichia coli infection

KEGGPATH

hsa05131:Shigellosis