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Human telomerase reverse transcriptase mRNA editing (introduction of HSVtk sequence)

Molecular classification
mRNA, Gene therapy target
01

Overview

Human telomerase reverse transcriptase (hTERT) is the catalytic subunit of telomerase, essential for maintaining telomere length and enabling cellular immortality, particularly in cancer cells. Editing hTERT mRNA to introduce the herpes simplex virus thymidine kinase (HSVtk) sequence allows for targeted cancer therapy. Cells expressing the edited mRNA produce HSVtk, which converts the prodrug ganciclovir into a toxic metabolite, leading to selective cell death. This approach aims to specifically eliminate cancer cells based on their hTERT expression.

02

Mechanism of action

Upon expression of HSVtk, ganciclovir is converted into a toxic form that inhibits DNA replication and induces cell death.

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Biological functions

Telomere maintenance (when unedited)Cell death (when edited with HSVtk)
04

Disease associations

Cancer
05

Safety considerations

Off-target effects of gene editingImmune response to HSVtkPotential for insertional mutagenesis
06

Interacting drugs

Ganciclovir
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Biomarkers

hTERT expression level

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