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ASAH1 shRNA
- Development stage
- Preclinical
- Lead developer
- University of Alabama at Birmingham
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Parenteral
01
Overview
ASAH1 shRNA is a genetic research tool designed to silence the expression of acid ceramidase (ASAH1), an enzyme that plays a critical role in sphingolipid metabolism by hydrolyzing ceramides into sphingosine. In glioblastoma (GBM), ASAH1 is frequently overexpressed, leading to a shift in the sphingolipid balance toward sphingosine-1-phosphate (S1P), which promotes tumor cell survival, migration, and resistance to chemotherapy. By utilizing short hairpin RNA (shRNA) to knockdown ASAH1 expression, researchers aim to restore the sphingolipid balance, increasing pro-apoptotic ceramide levels and decreasing pro-survival S1P levels. Preclinical studies in patient-derived xenograft models have demonstrated that ASAH1 knockdown can inhibit tumor growth and migration, particularly in the context of temozolomide (TMZ) resistance.
- Other names
- acid ceramidase shRNAshRNA targeting ASAH1
02
Targets
Acid ceramidase 1 mRNAASAH1 (Acid ceramidase)
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