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.A new method called RENDER enables transient delivery of epigenome editors into human cells. This approach overcomes size limitations of CRISPR-based tools, facilitating gene expression modulation for research and potential therapies.
Area Of Science
- Molecular Biology
- Epigenetics
- Gene Editing Technologies
Background
- Programmable epigenome editors alter gene expression by modifying chromatin without DNA breaks.
- CRISPR-based epigenome editors face delivery challenges due to their large size, limiting their application in research and therapy.
- Efficient delivery systems are crucial for advancing epigenome editing applications.
Purpose Of The Study
- To develop a novel delivery platform for transiently introducing programmable epigenome editors into human cells.
- To overcome the size limitations associated with traditional CRISPR-based epigenome editing systems.
- To demonstrate the efficacy of the new platform in modulating gene expression and its potential therapeutic applications.
Main Methods
- Development of Robust ENveloped Delivery of Epigenome-editor Ribonucleoproteins (RENDER) platform.
- Delivery of ribonucleoprotein complexes containing epigenetic repressors (CRISPRi, DNMT3A-3L-dCas9, CRISPRoff) and activators (TET1-dCas9).
- Application in various human cell types, including primary T cells and stem cell-derived neurons.
Main Results
- RENDER enables transient delivery of epigenome editors as ribonucleoprotein complexes.
- Demonstrated durable epigenetic silencing of endogenous genes in diverse human cell types.
- Successfully applied RENDER to repress neurodegenerative disease-associated Tau protein in human stem cell-derived neurons.
Conclusions
- The RENDER platform provides an effective method for delivering CRISPR-based epigenome editors into human cells.
- RENDER overcomes size limitations, broadening the scope of epigenome editing in fundamental research.
- This technology holds promise for future therapeutic applications, including neurodegenerative diseases.
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