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PU27

Development stage: Preclinical
Modality: MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics

Overview

PU27 is an experimental G-quadruplex-forming DNA oligonucleotide derived from the Pu27 sequence in the proximal promoter region of the c-MYC proto-oncogene, designed as an antineoplastic agent by stabilizing a transcription-inhibitory G-quadruplex and thereby suppressing c-MYC expression. Preclinical studies indicate that exogenous genomic c-MYC quadruplex DNA based on PU27 can selectively kill leukemia cells, induce extensive DNA damage and chromosomal abnormalities, and inhibit tumor cell proliferation, positioning it as a potential targeted therapy for c-MYC–driven malignancies.[3][5][7]

Targets

SOX2-G4 (SOX2 promoter G-quadruplex)telomeric G4 (Telomeric DNA G-quadruplex)

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