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posted by Fnord666 on Monday July 15 2019, @12:14PM   Printer-friendly
from the crispier dept.

Submitted via IRC for AnonymousLuser

Novel nanoparticles deliver CRISPR gene editing tools into the cell with much higher efficiency

A research collaboration between Tufts University and the Chinese Academy of Sciences has led to the development of a significantly improved delivery mechanism for the CRISPR/Cas9 gene editing method in the liver, according to a study published recently in the journal Advanced Materials. The delivery uses biodegradable synthetic lipid nanoparticles that carry the molecular editing tools into the cell to precisely alter the cells' genetic code with as much as 90 percent efficiency. The nanoparticles represent one of the most efficient CRISPR/Cas9 delivery tools reported so far, according to the researchers, and could help overcome technical hurdles to enable gene editing in a broad range of clinical therapeutic applications.

The CRISPR/Cas9 gene editing system has become a powerful research tool uncovering the function of hundreds of genes and is currently being explored as a therapeutic tool for the treatment of various diseases. However, some technical hurdles remain before it can be practical for clinical applications. CRISPR/Cas9 is a large molecular complex, containing both a nuclease (Cas9) that can cut through both strands of a targeted genomic sequence, and an engineered 'single-guide' RNA (sgRNA) that scans the genome to help the nuclease find that specific sequence to be edited. Since it is a large molecular complex, it is difficult to deliver CRISPR/Cas9 directly into the nucleus of the cell, where it can do its work. Others have packed the editing molecules into viruses, polymers, and different types of nanoparticles to get them into the nucleus, but the low efficiency of tranfer[sic] has limited their use and potency for clinical applications.

The lipid nanoparticles described in the study encapsulate messenger RNA (mRNA) encoding Cas9. Once the contents of the nanoparticles—including the sgRNA—are released into the cell. The cell's protein-making machinery takes over and creates Cas9 from the mRNA template, completing the gene editing kit. A unique feature of the nanoparticles is made of synthetic lipids comprising disulfide bonds in the fatty chain. When the particles enter the cell, the environment within the cell breaks open the disulfide bond to disassemble the nanoparticles and the contents are quickly and efficiently released into the cell.


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  • (Score: 2) by ikanreed on Monday July 15 2019, @03:09PM (4 children)

    by ikanreed (3164) Subscriber Badge on Monday July 15 2019, @03:09PM (#867206) Journal

    That's a virus. You're describing a virus.

    Starting Score:    1  point
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    Total Score:   2  
  • (Score: 0) by Anonymous Coward on Monday July 15 2019, @05:14PM (3 children)

    by Anonymous Coward on Monday July 15 2019, @05:14PM (#867244)

    Yeah, but it's *our* virus, not some random virus!

    The real question:
    When can I has the shot so I don't get sick no more?

    • (Score: 2) by ikanreed on Monday July 15 2019, @05:18PM (2 children)

      by ikanreed (3164) Subscriber Badge on Monday July 15 2019, @05:18PM (#867245) Journal

      They invented that, it's called potassium cyanide.

      • (Score: 0) by Anonymous Coward on Monday July 15 2019, @05:24PM (1 child)

        by Anonymous Coward on Monday July 15 2019, @05:24PM (#867250)

        You first, life isn't that bad yet (for me).

        • (Score: 2) by ikanreed on Monday July 15 2019, @05:37PM

          by ikanreed (3164) Subscriber Badge on Monday July 15 2019, @05:37PM (#867256) Journal

          Hey, I was just offering solution to the problem as stated.