Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
WT1-AS shRNA
- Development stage
- Preclinical
- Lead developer
- Technische Universität Dresden
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Parenteral, Intratumoral, Intralesional
01
Overview
WT1-AS shRNA is a short hairpin RNA designed to silence the long non-coding RNA (lncRNA) WT1-AS. WT1-AS is highly expressed in leukemic stem cells (LSCs) and acts as a regulator of stemness and a suppressor of myeloid differentiation in acute myeloid leukemia (AML). By knocking down WT1-AS, the shRNA induces cell cycle arrest in the G0/G1 phase and promotes apoptosis by reintroducing myeloid differentiation programs and downregulating stemness-associated genes. Preclinical studies in AML cell lines and patient-derived xenograft (PDX) models have demonstrated that WT1-AS knockdown reduces AML engraftment and progression, highlighting its potential as a therapeutic target for chemoresistant leukemic cells.
- Other names
- WT1-AS knockdownWT-1-AS knockdownWT 1-AS knockdownshRNA-based WT1-AS knockdown
Beyond the preview
Go deeper on WT1-AS shRNA.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on WT1-AS shRNA.