Drug intelligence / Profile preview

gPNA D-NLS

Development stage
Preclinical
Lead developer
University of Connecticut
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptides, 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
Administration
Intravenous
01

Overview

gPNA D-NLS is a synthetic oligonucleotide conjugate designed for targeted gene modulation in cancer. It consists of a gamma-modified peptide nucleic acid (gPNA) sequence targeting the c-Myc oncogene, conjugated to a nuclear localization signal (NLS) composed of D-amino acids (D-NLS). The D-amino acid modification enhances the proteolytic stability of the NLS in human serum compared to standard L-amino acid versions, while maintaining efficient nuclear transport via the importin alpha pathway. By targeting the c-Myc genomic DNA, the gPNA component achieves potent gene knockdown (approximately 90% in U2932 cells), leading to reduced protein levels and decreased cell viability. This technology is being developed at the University of Connecticut for potential applications in cancer treatment and other genetic modulation strategies.

Other names
gPNA D-NLS conjugategammaPNA-NLS construct
02

Targets

Nuclear import machineryMYC (MYC proto-oncogene protein)

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