Carnegie Mellon / Yale PNA-based technology is an important part of the gene editing toolkit

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In an article published in the April 8 issue of NatureThe National Institutes of Health’s Somatic Gene Editing Consortium nationwide edits the genome of disease-related somatic cells to develop safer and more effective ways to reduce the burden of diseases caused by genetic alterations. We have provided detailed and up-to-date information on the progress of our efforts.

Genetic editing allows scientists to modify the section of DNA in an organism and is considered a promising treatment for many genetic disorders. Although many advances have been made in the laboratory over the last few decades, there are still many challenges that must be overcome before gene editing can be widely used in patient populations. Launched in 2018, the Somatic Cell Gene Editing Consortium (SCGE) brings together some of the key researchers in the field to advance their discoveries and translate the progress of somatic gene editing in the laboratory into clinical settings. It accelerated.

NIH will allocate approximately $ 190 million to SCGE to realize gene editing potential over a six-year period. The end result will be a free-to-use toolkit that provides the biomedical research community with rigorously evaluated information on methods for delivering and tracking genome editors and gene-editing molecules.

“NIH realized that it was important for all of us investigating gene editing to work together toward a common goal,” said Daniel Ly, a professor of chemistry at Carnegie Mellon University who joined the consortium in 2019. Says. Cell and we catalog each and every one. What we ultimately get is a very valuable and rigorously valued resource for anyone who wants to bring gene editing to their patients. “

While much of the consortium’s work has focused on CRISPER-Cas related systems, SCGE points out that it is important to continue developing other systems. They specifically selected a peptide nucleic acid-based gene editing technique developed by Ly at Carnegie Mellon University and Peter Glazer at Yale University.

“Although the focus is heavily on CRISPR-Cas-related systems within the SCGE, alternative systems may continue to be investigated as both delivery potential and biological or immunological responses can differ. It’s important, “the consortium wrote.To Nature..

CRISPR-Cas edits the genes of cells that have been removed from the body, while Ly and Glazer’s Peptide Nucleic Acid (PNA) system is administered intravenously and edits cells in vivo. Nanoparticles are used to deliver PNA molecules paired with DNA donor strands directly to dysfunctional genes. Ly, a leader in synthetic nucleic acid technology, has programmed the PNA molecule to open double-stranded DNA at the site of the targeted mutation. Donor DNA from the complex binds to defective DNA in cells and triggers the innate repair mechanism of DNA to edit the gene. The team used this technique to treat beta-thalassemia in adult and fetal mice in utero.

The PNA gene editing system does not have the high yields of the CRISPER-Cas system, but it has the advantage of being less likely to make off-target changes. According to Ly, that means their technique may be better for genetic disorders that require only a small percentage of cells to be modified to make a therapeutic difference. For example, in a beta thalassemia study, Ly and Glazer found that editing only 6 to 7 percent of cells was curative.

Ly and Glazer plan to further refine and improve their technology by joining SCGE and look forward to sharing the results with the consortium and the larger biomedical community.

Genome editing to treat human retinal degeneration

For more information:
NIH Somatic Genome Editing Program, Nature (2021). DOI: 10.1038 / s41586-021-03191-1

Courtesy of Carnegie Mellon University

Quote: Carnegie Mellon / Yale PNA-based technology is an important part of the Gene Editing Toolkit (2021, April 7), https: // Obtained from based- on April 7, 2021. Technique-essential.html

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Carnegie Mellon / Yale PNA-based technology is an important part of the gene editing toolkit

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