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interleukin-1 receptor accessory protein short hairpin RNA
- Development stage
- Preclinical
- Lead developer
- Albert Einstein College of Medicine
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
Interleukin-1 receptor accessory protein (IL1RAP) short hairpin RNA is an experimental RNA interference (RNAi) therapeutic designed to silence the expression of the IL1RAP gene. IL1RAP is a co-receptor for the interleukin-1 receptor and is highly expressed on the surface of leukemic stem cells in acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndromes (MDS). By reducing IL1RAP levels, this shRNA inhibits downstream inflammatory signaling and disrupts the amplification of FLT3 and c-KIT pathways, which are critical for the survival and proliferation of leukemic stem cells. Research conducted at the Albert Einstein College of Medicine has demonstrated that targeting IL1RAP with shRNA induces cell death in AML cells while sparing normal hematopoietic stem cells.
- Other names
- IL1RAP shRNAIL-1RAP shRNAIL 1RAP shRNAshRNA targeting IL1RAP
02
Targets
IL1RAP (Interleukin 1 receptor accessory protein)
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