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Updated: Jul 1, 2026
09:51
Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
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Delivery of Prime editing in human stem cells using pseudoviral NanoScribes particles
Thibaut Halegua1, Valérie Risson2, Julien Carras2,3
1CIRI, Centre International de Recherche en Infectiologie Univ Lyon, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, ENS de Lyon, F-69007, Lyon, France.
Nature Communications
|January 4, 2025
Summary
Researchers developed Nanoscribes, virus-like particles for precise gene editing in human stem cells. This breakthrough overcomes delivery challenges for prime editing, enabling advanced genome engineering applications.
Area of Science:
- Molecular Biology
- Gene Editing Technologies
- Biotechnology
Background:
- Prime editing enables precise DNA modifications like point mutations, deletions, and insertions.
- Efficient and safe delivery of prime editing tools into human stem cells is a significant challenge.
Purpose of the Study:
- To engineer Nanoscribes, virus-like particles for delivering prime editing ribonucleoprotein complexes into cells.
- To optimize Nanoscribes for enhanced efficiency, fidelity, and broad applicability in human stem cells.
Main Methods:
- Engineering virus-like particles (Nanoscribes) encapsulating prime editing components.
- Optimizing Nanoscribe features including fusogens, pegRNA structures, Pol II encoding, and prime editor variants.
- Assessing editing efficiency and fidelity in HEK293T cells, myoblasts, hiPSCs, and hiPSC-derived hematopoietic stem cells.
Main Results:
- Nanoscribes achieved 68% editing efficiency at the HEK3 locus in HEK293T cells with high fidelity.
- Demonstrated successful editing in primary human cells, including myoblasts, hiPSCs, and hematopoietic stem cells, with up to 25% efficiency.
- Nanoscribes support pegRNA multiplexing and show improved fidelity compared to DNA transfection.
Conclusions:
- Nanoscribes represent a novel and efficient delivery system for prime editing in various human cell types.
- This VLP-based technology advances the development of next-generation genome editing strategies.
- Nanoscribes overcome key delivery hurdles, paving the way for therapeutic applications of precise gene editing.
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