Target intelligence / Profile preview

Cytosolic carboxypeptidase 1 (AGTPBP1)

Target
AGTPBP1
Molecular classification
Enzyme, Metallocarboxypeptidase, Protein deglutamylase
01

Overview

Cytosolic carboxypeptidase 1 (AGTPBP1) is a zinc-dependent metallocarboxypeptidase involved in the removal of polyglutamate side chains from the gamma-carboxyl group of glutamate residues, particularly within the C-terminal tail of alpha- and beta-tubulin, as well as certain non-tubulin proteins[1][2][3][6][7]. This posttranslational deglutamylation modulates microtubule stability, axonal maintenance, and neural function. Mutations in AGTPBP1 cause notable neurodegenerative phenotypes in mice (Purkinje cell degeneration) and humans (childhood-onset cerebellar atrophy), implicating this enzyme in normal neuronal survival and cytoskeletal regulation[3][4]. The protein contains a zinc carboxypeptidase domain and nuclear localization signals, and was originally identified in regenerating neurons[1][6][7]. *Note: No approved drugs or validated clinical biomarkers are known to specifically target AGTPBP1 as of the current evidence. Mechanisms of drug action and biomarker roles are not described in available primary sources.*

Other names
ATP/GTP binding carboxypeptidase 1CCP1KIAA1035NNA1ATP/GTP-binding protein 1Nervous system nuclear protein induced by axotomy protein 1 homologProtein deglutamylase CCP1Tubulinyl-Tyr carboxypeptidaseTyrosine carboxypeptidaseCarboxypeptidase-tubulinSoluble carboxypeptidaseCONDCA
02

Biological functions

Protein deglutamylation, especially of alpha- and beta-tubulin and non-tubulin proteinsModulation of microtubule structure and dynamicsRegulation of nervous system developmentNegative regulation of cell pluripotency maintenance and embryogenesis via KLF4 deglutamylation
03

Disease associations

Neurodegenerative disease (notably, childhood-onset neurodegeneration with cerebellar atrophy)Pontocerebellar hypoplasiaMotor coordination disorders (ataxia)Retinal photoreceptor degeneration
04

Safety considerations

Loss-of-function is associated with progressive Purkinje cell degeneration, ataxia, and neuronal loss, raising concerns for CNS toxicity if inhibited or impaired[1][3][4][6][7].

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