Target intelligence / Profile preview

PiggyBac transposable element-derived protein 1 (PGBD1)

Target
PGBD1
Molecular classification
Transcription factor (SCAN domain), DNA-binding protein, Other (KRAB-domain containing protein), PiggyBac transposase-derived protein (domesticated transposase, no longer enzymatic)
01

Overview

PiggyBac transposable element-derived protein 1 (PGBD1) is a mammal-specific, domesticated transposase-derived protein that has lost its ancestral transposase activity but gained regulatory functions through acquisition of SCAN and KRAB domains[1][2][3]. It acts as a DNA-binding transcriptional regulator, highly expressed in certain brain regions, and is involved in the maintenance and fate control of neuronal progenitor cells by controlling the transcription of the long non-coding RNA NEAT1, which is essential for paraspeckle formation[1][2][3]. PGBD1 binding modulates transcriptional pausing at target genes, especially NEAT1, and its depletion enhances NEAT1 expression, paraspeckle formation, and neuronal differentiation[1][2][3]. Genome-wide association studies have suggested that genetic variants in PGBD1 may influence schizophrenia risk, although findings are population-specific and not yet fully established[3]. PGBD1 is not known to be a direct therapeutic target, there are no known approved drugs or biomarker applications, and safety concerns related to direct pharmacological targeting have not been reported[1][2][3].

Other names
HUCEP-4SCAND4dJ874C20.4cerebral protein 4piggyBac transposable element-derived protein 1
02

Biological functions

Transcriptional regulationMaintenance of neuronal progenitor cellsRegulation of paraspeckle formation (via NEAT1 lncRNA)Regulation of neuronal development
03

Disease associations

Neurodevelopmental diseasePsychiatric disease (schizophrenia risk locus in some population studies)Other (potential brain function/dysregulation)

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