Drug intelligence / Profile preview

PDK1 shRNA

Development stage
Preclinical
Lead developer
University of Tübingen
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

PDK1 shRNA is a gene-silencing therapeutic approach designed to inhibit the expression of 3-phosphoinositide-dependent protein kinase 1 (PDK1). PDK1 is a master regulator of the PI3K/AKT signaling pathway, specifically responsible for the phosphorylation of AKT at the Thr308 residue. In the context of acute leukemia, PDK1-mediated AKT activation is crucial for the maintenance and proliferation of leukemic blasts, particularly within the protective bone marrow microenvironment. By utilizing retroviral vectors to deliver shRNA constructs, researchers can suppress PDK1 transcription, leading to diminished proliferation and reduced tumor engraftment in preclinical models of acute myeloid leukemia (AML).

Other names
PDK1-shRNAPDK-1-shRNAPDK 1-shRNAshort hairpin RNA targeting PDK1
02

Targets

PDPK1 (PDK1 / PDPK1 (PI3K-downstream))

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