Programmable epigenome editing by transient delivery of CRISPR epigenome editor ribonucleoproteins
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View abstract on PubMed
Summary
This summary is machine-generated.We developed a new method called RENDER to deliver epigenome editors into human cells. This system enables durable gene silencing and repression of disease-associated proteins, advancing epigenome editing for research and therapy.
Area Of Science
- Molecular Biology
- Gene Editing
- Epigenetics
Background
- Programmable epigenome editors alter gene expression without DNA breaks.
- CRISPR-based editors are large, limiting their application.
- Efficient delivery of epigenome editors is crucial for research and therapy.
Purpose Of The Study
- To develop a novel delivery platform for epigenome editors.
- To enable transient delivery of repressors and activators.
- To overcome the size limitations of current CRISPR-based systems.
Main Methods
- Developed Robust ENveloped Delivery of Epigenome-editor Ribonucleoproteins (RENDER).
- Engineered ribonucleoprotein complexes for transient delivery.
- Applied RENDER to deliver CRISPRi, DNMT3A-3L-dCas9, CRISPRoff, and TET1-dCas9.
Main Results
- RENDER achieved durable epigenetic silencing of endogenous genes in diverse human cell types, including primary T cells.
- Successfully repressed target genes in human stem cell-derived neurons.
- Reduced expression of the V337M-mutated Tau protein associated with neurodegenerative disease.
Conclusions
- RENDER is an advanced platform for delivering epigenome editors into human cells.
- This technology broadens the scope of epigenome editing in fundamental research.
- RENDER holds potential for future therapeutic applications.
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