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CRISPR-Cas9 ribonucleoprotein lipid nanoparticle
- Development stage
- Preclinical
- Lead developer
- Indiana 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 ribonucleoprotein lipid nanoparticle (RNP-LNP) is a gene-editing platform that delivers the Cas9 enzyme pre-complexed with guide RNA (gRNA) as a functional ribonucleoprotein unit encapsulated within lipid nanoparticles. This delivery format allows for immediate gene editing upon cellular entry and rapid clearance of the editing machinery, potentially minimizing off-target effects compared to DNA or mRNA delivery. A specific application developed at Indiana University involves "exosome-inspired" LNPs that mimic the lipid and protein composition of lung epithelial-derived exosomes to achieve preferential targeting of long-term hematopoietic stem cells (LT-HSCs) in the bone marrow. This platform is being investigated for the in vivo treatment of β-hemoglobinopathies, such as sickle cell disease and thalassemia, by targeting the BCL11A erythroid enhancer to induce fetal hemoglobin expression.
- Other names
- exosome-inspired CRISPR LNPsCRISPR RNP-LNP
02
Targets
Hemoglobin subunit gamma-1 promoterBCL11A (B-cell CLL/lymphoma 11A)CCR5 (C-C chemokine receptor type 5)
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