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siRNA-BRAF V600E
- Development stage
- Preclinical
- Lead developer
- Brigham and Women's Hospital
- 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
01
Overview
siRNA-BRAF V600E is a target-specific small interfering RNA (siRNA) designed to selectively silence the mutated BRAF V600E allele. The BRAF V600E mutation leads to constitutive activation of the MAPK/ERK signaling pathway, driving aberrant cell proliferation and survival in various cancers and histiocytic disorders. By targeting the mutated mRNA sequence, siRNA-BRAF V600E reduces the expression of the mutant BRAF protein, thereby restoring normal cellular phenotypes. It has been investigated preclinically for the treatment of Langerhans cell histiocytosis (LCH) with bone involvement, where it aims to normalize hyperactive osteoclastogenesis, as well as in melanoma models.
- Other names
- BRAF (V600E) siRNABRAF V600E siRNAsiRNA-Braf V600EsiRNA-Braf-V600EsiRNA-Braf-V-600EsiRNA-Braf-V 600E
02
Targets
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