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siRNA against REST
- Development stage
- Preclinical
- 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
- Intrathecal, Intratumoral
01
Overview
siRNA against REST (siREST) is a preclinical-stage RNA interference (RNAi) therapeutic designed to silence the RE1-Silencing Transcription Factor (REST), also known as Neuron-Restrictive Silencer Factor (NRSF). REST is a transcriptional repressor that is abnormally upregulated in various neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), and certain cancers like glioblastoma. By targeting and degrading REST mRNA, this siRNA-based agent aims to restore the expression of essential neuronal genes, mitigate motor neuron loss, and reduce protein aggregation. In preclinical models, delivery of REST-targeting siRNA has shown therapeutic potential in extending survival in ALS models and inhibiting tumor progression in glioblastoma.
- Other names
- RE1-silencing transcription factor small interfering RNARE-1-silencing transcription factor small interfering RNARE 1-silencing transcription factor small interfering RNAREST siRNAsiREST
02
Targets
REST (Repressor element-1 silencing transcription factor)
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