Researchers from the Gladstone Institute and the University of California, San Francisco have developed a new
The CRISPR-Cas9 gene editing system showedits effectiveness in many studies. It allows you to turn off, delete or replace individual sections of genes. The technology has traditionally used viral vectors to transfer the necessary DNA (matrix) - the shells of viruses without their pathogenic components. A key drawback of the technology is that it is difficult for scientists to control exactly where the right material will be inserted, which led to multiple side mutations.
In their work, scientists use single-strandedDNA and the same Cas9 protein, which acts like scissors, cutting the molecule in the right places. Back in 2015, the authors of the work demonstrated for the first time the possibility of inserting short DNA templates into immune cells without viral vectors using an electric field. New work has combined this technology using the Cas9 protein.
The study showed that the new method of geneediting simplifies the reengineering of large numbers of cells for therapeutic purposes. In this case, the efficiency reaches 80-90%. As a result, scientists were able to quickly and efficiently generate enough CAR-T cells (genetically modified T cells).
The created cells can be used to fight againstmultiple myeloma, blood cancer, and to rewrite the sequence of genes whose mutations can lead to rare hereditary immune diseases.
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