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CRISPR-Cas9 lipid nanoparticle targeting APOE4
- Development stage
- Preclinical
- Lead developer
- Columbia University
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
CRISPR-Cas9 lipid nanoparticle targeting APOE4 is an experimental gene-editing therapeutic designed to treat Alzheimer's disease by reducing the expression of the apolipoprotein E ε4 (APOE4) allele, the strongest genetic risk factor for the condition. Developed by researchers at Columbia University, this nonviral platform utilizes lipid nanoparticles (LNPs) to deliver CRISPR-Cas9 components specifically to the brain. To overcome the blood-brain barrier, the treatment is administered systemically in conjunction with focused ultrasound (FUS)-mediated blood-brain barrier opening. Preclinical studies in transgenic mice have demonstrated that this approach can achieve significant APOE4 knockdown, leading to reduced amyloid-beta accumulation and attenuated neuroinflammatory responses, such as decreased astrocyte and microglial activation.
- Other names
- CRISPR-Cas9 LNPs targeting APOE4CRISPR-Cas-9 LNPs targeting APOE4CRISPR-Cas 9 LNPs targeting APOE4nonviral CRISPR therapeutics targeting APOE4
02
Targets
APOE4 (Apolipoprotein E4)
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