Drug intelligence / Profile preview

PTEN shRNA

Development stage
Preclinical
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral, Intravenous, Ophthalmic, Intravitreal, Inhalation, Oral
01

Overview

PTEN shRNA is a short hairpin RNA designed to silence the expression of the Phosphatase and tensin homolog (PTEN) gene through RNA interference (RNAi). PTEN is a critical tumor suppressor that acts as a phosphatase to dephosphorylate PIP3, thereby negatively regulating the PI3K/AKT/mTOR signaling pathway. In oncological research, PTEN shRNA is frequently employed—often delivered via lentiviral or adenoviral vectors—to model the loss of PTEN function observed in various cancers, including melanoma, prostate cancer, and glioblastoma. Knockdown of PTEN leads to constitutive activation of pro-survival and proliferative pathways; for instance, research indicates that PTEN depletion in melanocytes can upregulate EPAC-RAP1 signaling, facilitating malignant transformation and senescence escape.

Other names
PTEN-KDPTEN short hairpin RNA
02

Targets

PTEN (Phosphatase and tensin homolog deleted on chromosome ten)

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