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SRSF1-shRNA
- Development stage
- Preclinical
- Lead developer
- Soochow University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
SRSF1-shRNA is a short hairpin RNA (shRNA) therapeutic candidate designed to silence the expression of Serine/arginine-rich splicing factor 1 (SRSF1), also known as ASF/SF2. SRSF1 is a proto-oncogenic splicing factor that is frequently overexpressed in multiple myeloma (MM) and other hematological malignancies, where it promotes tumorigenesis by modulating the alternative splicing of genes involved in cell cycle regulation and apoptosis, such as RBBP6. By silencing SRSF1, the shRNA shifts splicing patterns from oncogenic isoforms to tumor-suppressive ones, leading to inhibited cell proliferation, increased programmed cell death, and cell cycle arrest. Preclinical evidence from orthotopic xenograft models suggests that SRSF1-shRNA can significantly reduce tumor burden and extend survival in multiple myeloma.
- Other names
- sh-SRSF1sh-SRSF-1sh-SRSF 1SRSF1 short hairpin RNASRSF-1 short hairpin RNASRSF 1 short hairpin RNA
02
Targets
SRSF1 (Serine/arginine-rich splicing factor 1)
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