Drug intelligence / Profile preview

shTFAM

Development stage
Unknown
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

shTFAM is a short hairpin RNA (shRNA) construct designed to silence the expression of Transcription Factor A, Mitochondrial (TFAM). TFAM is a critical nuclear-encoded protein that regulates the transcription and replication of mitochondrial DNA (mtDNA). In the context of gastrointestinal research, shTFAM is used as an experimental tool to model mitochondrial dysfunction in human colonoids and intestinal epithelial cells. By reducing TFAM levels, researchers can induce a state of mitochondrial deficiency characterized by lower mtDNA-encoded gene expression (e.g., Cox6A1, mt-CO1, ATP5A1) and reduced oxygen consumption rates. This model has been used to demonstrate that mitochondrial deficiency impairs crypt fissioning and mucosal repair in Inflammatory Bowel Disease (IBD), suggesting that therapeutic strategies to improve mitochondrial function could enhance ulcer healing.

Other names
TFAM shRNAshort hairpin RNA targeting TFAM
02

Targets

Transcription factor A, mitochondrial mRNA

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