Drug intelligence / Profile preview

shRNA-DDIT4

Development stage
Unknown
Lead developer
Origene Technologies
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral, Intravitreal, Intrathecal, Intramuscular
01

Overview

shRNA-DDIT4 refers to short hairpin RNA (shRNA) constructs designed to silence the expression of the DNA Damage Inducible Transcript 4 (DDIT4) gene, also known as REDD1 (Regulated in Development and DNA Damage Response 1). DDIT4 is a potent negative regulator of the mechanistic target of rapamycin complex 1 (mTORC1) pathway, typically induced under stress conditions such as hypoxia, oxidative stress, or DNA damage. By utilizing RNA interference (RNAi) to degrade DDIT4 mRNA, these shRNA molecules effectively disinhibit mTORC1 signaling. This mechanism is of significant therapeutic interest for conditions characterized by pathological mTORC1 suppression, including neurodegenerative diseases like Parkinson's disease, skeletal muscle atrophy, and certain retinal disorders such as diabetic retinopathy and age-related macular degeneration. While primarily utilized as a research tool to investigate cellular stress responses and mTOR regulation, shRNA-DDIT4 constructs are explored in preclinical models as potential gene therapy candidates for neuroprotection and tissue regeneration.

Other names
REDD1 shRNAREDD-1 shRNAREDD 1 shRNARTP801 shRNARTP-801 shRNARTP 801 shRNAshDDIT4shDDIT-4shDDIT 4
02

Targets

AGO2/RISC (Protein Argonaute-2 (AGO2) and RNA-induced silencing complex (RISC))DDIT4 (Regulated in development and DNA damage response protein 1)

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