Target intelligence / Profile preview

Purine-rich element-binding protein A (PURA)

Target
PURA
Molecular classification
Transcription factor, Nucleic acid-binding protein, Single-stranded DNA-binding protein, RNA-binding protein, Whirly-like nucleic acid-binding family
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Overview

Purine-rich element-binding protein A (PURA) is a highly conserved, sequence-specific single-stranded DNA- and RNA-binding protein best known for its regulatory roles in gene transcription and mRNA localization. It binds preferentially to purine-rich single-stranded DNA or RNA and is involved in the unwinding of double-stranded nucleic acids in an ATP-independent manner, which is critical for nucleic acid structural remodeling. This protein is a member of the ancient "Pur" family, with a structural fold known as a Whirly-like domain, mediating both nucleic acid binding and protein-protein interactions. PURA plays diverse roles in neurodevelopment, and mutations in the PURA gene cause PURA syndrome, a neurodevelopmental disorder marked by hypotonia, developmental delay, and other neurological deficits. PURA has also been implicated in viral replication, such as HIV, by binding viral RNAs. There are no established pharmacological agents that target PURA directly, although it is a potential therapeutic and diagnostic target in neurological and viral diseases.

Other names
Transcriptional activator protein Pur-alphaPUR1PURALPHAPUR-ALPHAPurine-rich single-stranded DNA-binding protein alphaMRD31NEDRIHF
02

Mechanism of action

No drugs approved that act through direct modulation; thus, mechanism of drug action on this target is not established.

03

Biological functions

Regulation of transcriptionRNA localizationDNA and RNA binding (single-stranded)Unwinding of double-stranded nucleic acids (ATP-independent)Regulation of gene expressionProtein-protein interaction mediatorNeuroprotection
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Disease associations

Neurodevelopmental disorders (notably PURA syndrome)Neurological diseasesPotential host factor for viral propagation (e.g., HIV)Other roles in rare inherited diseases
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Safety considerations

Targeting a fundamental nucleic acid-binding protein involved in widespread regulation poses high risk of off-target or systemic toxicitiesMutations can cause widespread neurodevelopmental deficits
06

Biomarkers

PURA gene mutation (notably as a diagnostic marker for PURA syndrome and some neurodevelopmental disorders)

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