Transcriptional activator protein Pur-alpha (Pur-alpha)

Transcriptional activator protein Pur-alpha (Pur-alpha) is a highly conserved, multifunctional DNA- and RNA-binding protein belonging to the PUR family (UniProt: Q00577). It plays a pivotal role in regulating gene transcription, DNA replication, and RNA transport, particularly within the central nervous system (PubMed: 20976240). Pur-alpha binds to purine-rich sequences (PUR elements) to modulate the expression of various genes, such as MYC and myelin basic protein (MBP), and is essential for normal brain development and neuronal maturation (MedlinePlus). Mutations or deletions in the PURA gene are the primary cause of PURA syndrome and 5q31.3 microdeletion syndrome, which manifest as severe neurodevelopmental delays, hypotonia, and epilepsy (PubMed: 25434005). In neurodegenerative conditions like amyotrophic lateral sclerosis (ALS) and fragile X-associated tremor/ataxia syndrome (FXTAS), Pur-alpha is sequestered into pathological RNA foci, contributing to disease progression (PubMed: 23933751). Additionally, Pur-alpha is implicated in cancer progression and the replication of viruses such as HIV-1 and JC virus (PubMed: 11158610). Current therapeutic research explores small molecules, such as G699-0288, to modulate its protein-protein interactions or expression levels for treating cancer and neurodegenerative disorders (PubMed: 37504270).

Other names

PURAPUR1PUR-ALPHAPURALPHAMRD31NEDRIHFPurine-rich single-stranded DNA-binding protein alpha

Modulation of protein-protein interactions and transcriptional regulation to inhibit cancer progression or provide neuroprotection.