Target intelligence / Profile preview

RUN and SH3 domain containing 1 (RUSC1)

Target
RUSC1
Molecular classification
Adaptor protein, Cytoskeletal-associated protein, Other
01

Overview

RUN and SH3 domain containing 1 (RUSC1) is an adaptor protein with actin-binding properties, involved in protein polyubiquitination and vesicular trafficking. It associates with the AP-4 adaptor complex, influencing neuronal vesicle transport and differentiation, specifically via regulation of NGF-dependent neurite outgrowth and the MAPK and NF-κB pathways. While not a direct drug target, its antisense transcript RUSC1-AS1 modulates major cancer-related signaling pathways (Wnt/β-catenin, PI3K/AKT), contributing to tumor progression and serving as a potential cancer biomarker. RUSC1 mutations are linked to autosomal recessive hereditary spastic paraplegia.

Other names
NESCANescaAP-4 complex accessory subunit RUSC1RUN and SH3 domain-containing protein 1New molecule containing SH3 at the carboxy-terminus
02

Biological functions

Actin bindingProtein polyubiquitinationVesicular traffickingNGF-dependent neurite outgrowthRegulation of neuronal differentiationNF-κB pathway modulation
03

Disease associations

Hereditary spastic paraplegia (Spastic Paraplegia 50, Autosomal Recessive)Cancer (through antisense RNA RUSC1-AS1's regulatory roles in breast cancer and osteosarcoma)
04

Biomarkers

RUSC1-AS1

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