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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Nanocapsules show promise in delivering gene-editing tools to cells

Researchers at the University of Wisconsin–Madison have successfully developed a customizable nanocapsule that could deliver CRISPR-Cas9, a frequently used gene editing machinery for editing genes in vivo.

Most gene editing tools are delivered to cells using viral vectors. However, the safety concerns associated with viral vectors have prompted researchers to look for alternative delivery tools.

In the present study published in Nature Nanotechnology, Dr Shaoqin Gong, Dr Krishanu Saha and teams at the University of Wisconsin–Madison developed a customizable nanocapsule that could encapsulate the CRISPR-Cas9 gene editing machinery and deliver it into cells safely.

The study showed that the nanocpasule, 25nm in diameter, could be coated with specific ligands which would give them the ability to target specific cell types. The nanocapsules stay intact outside cells and inside the target cell, they break open by a molecule called glutathione to release the CRISPR-Cas9 editing machinery which then moves to the nucleus to edit the cell’s DNA.

Data showed that the synthesized nanocapsules efficiently generated targeted gene edits in vitro without any cytotoxicity. In addition, it led to successful gene editing in vivo in murine retinal pigment epithelium tissue and skeletal muscle after local administration.

One of the major advantages of nanocapsules is that they are expected not to make unintended gene edits due to their short lifespan inside cells. In addition, they can freeze-dried, conveniently purified, stored and transported as a powder, while providing flexibility for dosage control.

The study highlights nanocapsule as a promising platform for gene therapies. The team is now working to further optimize the nanocapsules for efficient gene editing in the brain and the eye.

Source: A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing. Chen G et al., Nature Nanotechnology, September 2019. Website

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