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MALAT1-siRNA
- Development stage
- Preclinical
- Lead developer
- Wayne State University
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
MALAT1-siRNA is a small interfering RNA (siRNA) designed to silence the expression of the long noncoding RNA (lncRNA) Metastasis Associated Lung Adenocarcinoma Transcript 1 (MALAT1). In the context of diabetic retinopathy, MALAT1 is upregulated in retinal endothelial cells under hyperglycemic conditions, leading to the hypermethylation of the Mfn2 promoter and subsequent mitochondrial dysfunction. By targeting MALAT1, this siRNA restores Mfn2 expression, improves mitochondrial dynamics, and protects against structural and functional damage in the retina. It is currently being investigated in preclinical models for its potential to treat diabetic retinopathy and other complications associated with mitochondrial instability.
- Other names
- si-MALAT1si-MALAT-1si-MALAT 1MALAT1 siRNAMALAT-1 siRNAMALAT 1 siRNA
02
Targets
MALAT1 (Metastasis associated lung adenocarcinoma transcript 1)
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