A new platform for gene editing has been created by a team of researchers.
The team used chemical design and synthesis to bring together the winning technology with the therapeutic technology they created in their own lab. The cargo needed for generating the CRISPR-Cas9 is provided by the new work. In order to access a broad range of tissue and cell types, as well as the intracellular compartments required for gene editing, the team developed a way to transform the Cas-9Protein into a spherical nucleic acid.
A paper titled "CRISPR Spherical Nucleic Acids" was published in the Journal of the American Chemical Society.
The work builds on the 25-year effort led by Chad A. Mirkin to uncover the properties of SNAs and the factors that distinguish them from their linear cousins. The chemical and physical properties of SNAs are vastly different from those found in nature.
Mirkin is a professor of chemistry in the Weinberg College of Arts and Sciences. He is a professor of engineering and medicine at the Feinberg School of Medicine.
There are many classes of SNAs, with different chemical compositions and sizes, and they are being evaluated as potent therapeutic agents in six human clinical trials.
Mirkin said that the novel nanostructures provide a path for researchers to broaden the scope of their work by dramatically expanding the types of cells and tissues they can deliver to. Privileged access to the skin, the brain, the eyes, the immune system, the GI tract, heart and lungs is provided by SNAs. Good things will follow when this type of access is combined with one of the most important innovations in the last 25 years.
Mirkin's team attached DNA strands to the surface of the core of the structure in order to create a new type of SNA. These SNAs were fused with peptides to control their ability to navigate compartmental barriers of the cell in order to maximize efficiency. These SNAs are similar to other classes of SNAs in that they enter cells without the use of transfection agents.
The research team included graduate students.
More information: Chi Huang et al, CRISPR Spherical Nucleic Acids, Journal of the American Chemical Society (2022). DOI: 10.1021/jacs.2c07913 Journal information: Journal of the American Chemical Society
All Rights Reserved © 2024
