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CRISPR-Cas9 Ido1 targeting
- Development stage
- Preclinical
- Lead developer
- Columbia University
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous, Oral, Parenteral
01
Overview
CRISPR-Cas9 Ido1 targeting refers to the experimental use of the CRISPR-Cas9 gene-editing system to disrupt or knock out the *IDO1* (indoleamine 2,3-dioxygenase 1) gene. In the context of oncology research, specifically acute myeloid leukemia (AML), this approach is employed to inhibit the synthesis of kynurenine, an oncometabolite that promotes leukemia progression through the Kyn-HTR1B-SAA-IDO1 axis. By targeting *Ido1*, researchers have demonstrated a dose-dependent suppression of AML growth in mouse and human cell models, highlighting IDO1 as a critical therapeutic target within the bone marrow niche. This genetic intervention effectively blocks the autocrine activation of the aryl hydrocarbon receptor (AHR) and the pro-inflammatory remodeling of the osteoblastic niche that supports leukemia cell proliferation.
- Other names
- CRISPR-Cas9-mediated Ido1 targetingCRISPR-Cas-9-mediated Ido1 targetingCRISPR-Cas 9-mediated Ido1 targetingIDO1 CRISPR-Cas9IDO-1 CRISPR-Cas9IDO 1 CRISPR-Cas9
02
Targets
IDO1 (Indoleamine 2,3-dioxygenase 1)DNA
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