New gene editing platform expands use of CRISPR gene editing

Discovery expands use of CRISPR gene editing

CRISPR RNP (CRISPR SNA in action: In complex with single-guideline RNA (red), CRISPR SNA can recognize and split a desired gene target (yellow)). The complex form of Cas9 protein and single guide RNA is referred to as ribonucleoprotein (RNP). Credit: To Mirkin Lab/Northwestern University

A team of Northwestern University researchers has created a new gene-editing platform that could benefit the future application of a nearly unlimited library of CRISPR-based therapies.

Using chemical design and synthesis, the team combined Nobel Prize-winning technology with therapeutic technology generated in their lab to overcome serious limitations of CRISPR. Specifically, the pioneering work provides a system to deliver the payload required to generate a gene-editing machine known as CRISPR-Cas9. The team developed a method for converting the Cas-9 protein into spherical nucleic acid (SNA) and loading it with important components as required for access to a wide variety of tissues and cell typesas well as the intracellular compartments required for gene editing.

The research was published today in a paper titled “CRISPR Spherical Nucleic Acids” in the publication Journal of the American Chemical Societyand demonstrates how CRISPR SNAs can be delivered via Cell membrane and in the nucleus while also retaining vital activity and gene-editing capabilities.

The work builds on a 25-year effort led by nanotechnology pioneer Chad A. Merkin, who led the study, to reveal the characteristics of SNAs and the factors that distinguish them from their well-known linear cousin, the life planner. He is best known for inventing SNAs, structures typically composed of spherical nanoparticles densely covered with DNA or RNA, giving them chemical and physical properties radically different from those forms of nucleic acids found in nature.

Mirkin Professor of Chemistry by George B. He is also Professor of Chemical, Biological, Biomedical Engineering, and Materials Science and Engineering in the McCormick School of Engineering and Professor of Medicine at Northwestern University Feinberg School of Medicine.

Many classes of SNAs exist, with cores and shells of different chemical compositions and sizes, and SNAs are now being evaluated as effective treatments in six human clinical trials, including trials for debilitating diseases such as glioblastoma multiforme (brain cancer) and a variety of skin cancers

“This novel nano It provides a path for researchers to extend the usefulness of CRISPR by exponentially expanding the types of cells and tissues that the CRISPR mechanism can reach. Eyes, immune system, digestive system, heart and lungs. When this kind of access is coupled with one of the most important innovations in biomedical science in the last quarter century, good things will follow.”

In this current research, Mirkin’s team used Cas9, a protein essential for gene editing, as the core of the structure, attaching DNA strands to its surface to make a new type of SNA. In addition, SNAs were preloaded with RNA capable of performing gene editing and combined with peptides to control their ability to navigate the partial barriers of the cell, thus increasing efficiency. SNAs, like other classes of SNAs, enter cells effectively without the use of transfection agents (which are often required to deliver genetic material into cells) and appear high gene editing Efficiency between 32% and 47% across several human and murine cell lines.

The research team included graduate student researchers Chi Huang, Zeno (Henry) Han, and Michael Evangelopoulos.

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more information:
Chi Huang et al., CRISPR, Spherical Nucleic Acids, Journal of the American Chemical Society (2022). DOI: 10.1021 / jacs.2c07913

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