Drug intelligence / Profile preview

c3 transferase

Development stage
Unknown
Lead developer
McKerracher
Modality
Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration
Direct Injection (intracerebral, Intraneural), Viral Vector Delivery, In Vitro Application
01

Overview

C3 transferase is a **bacterial exoenzyme** derived from *Clostridium botulinum*, known for its ability to irreversibly inhibit the small GTPase **RhoA** (and also RhoB and RhoC) via **ADP ribosylation** at asparagine 41[1][2][3]. This inhibition leads to cytoskeletal reorganization, stimulation of neurite and axonal outgrowth, and neuroprotection. C3 transferase is a protein of approximately 24 kDa, commonly used experimentally to inhibit RhoA signaling in models of central nervous system injury and Parkinson's disease[1][2][3]. Therapeutically, cell-permeable and viral vector formats have been studied, primarily for application in neural repair and regeneration, including spinal cord injury and experimental cell replacement therapy in Parkinson's disease[1][3]. Modified ("Cethrin") versions are in development for localized delivery to the CNS, with ongoing investigation into gene therapy and protein delivery approaches[1][3]. No major adverse effects have been observed in continuous expression or targeted delivery studies.

Brand names
Cethrin
Other names
exoenzyme c3 transferasec3c-3c 3c3 exoenzymec-3 exoenzymec 3 exoenzymec3 toxinc-3 toxinc 3 toxin
02

Targets

RHOB (Ras homolog family member B)RHOC (Ras homolog family member C)RHOA (Ras homolog gene family member A)

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