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PTEN plasmid
- Development stage
- Preclinical
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intratumoral
01
Overview
PTEN plasmid is an experimental gene therapy construct consisting of non-viral plasmid DNA encoding the Phosphatase and Tensin Homolog (PTEN) tumor suppressor gene. PTEN acts as a dual-specificity phosphatase that negatively regulates the PI3K/Akt/mTOR signaling pathway by dephosphorylating phosphatidylinositol (3,4,5)-trisphosphate (PIP3). Loss or mutation of PTEN is a frequent occurrence in various malignancies, including glioblastoma, prostate cancer, and melanoma, leading to constitutive Akt activation and uncontrolled cell growth. Delivery of the PTEN plasmid aims to restore functional PTEN expression, thereby inhibiting tumor proliferation, inducing apoptosis, and potentially overcoming resistance to other therapies. Preclinical research has explored various delivery systems, such as cationized gelatin hydrogel microspheres (GelaTen) and lipid nanoparticles (PL-NANO), to improve the stability and intratumoral release of the plasmid DNA.
- Other names
- PTEN plasmid DNAplasmid-encoded PTEN
02
Targets
PIP3 (Phosphatidylinositol (3,4,5)-trisphosphate)Insulin receptor substrate 1 and related adaptor proteinsdTMP-SC (Nuclear thymidylate synthesis complex)
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