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MAML1 shRNA
- Development stage
- Preclinical
- Lead developer
- Medical College of Georgia
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
MAML1 shRNA is a short hairpin RNA (shRNA) designed to silence the expression of Mastermind-like protein 1 (MAML1), a critical transcriptional co-activator in the Notch signaling pathway. In the context of stem cell leukemia-lymphoma (SCLL) driven by the ZNF198-FGFR1 fusion protein, constitutive Notch1 activation is a key driver of leukemogenesis. By knocking down MAML1, the assembly of the Notch transcriptional complex is disrupted, leading to reduced expression of downstream Notch targets. Preclinical studies in syngeneic mouse models have demonstrated that MAML1 shRNA can significantly delay or prevent the development of leukemia and lymphoma, suggesting that targeting the Notch co-activator complex is a potential therapeutic strategy for SCLL and related hematological malignancies.
- Other names
- MAML1 short hairpin RNAMAML-1 short hairpin RNAMAML 1 short hairpin RNAshMAML1shMAML-1shMAML 1
02
Targets
MAML1 (Mastermind-like protein 1)
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