Scientists sharpen gene editing tool

Credit: CC0 Public Domain

Wake Forest Institute for Regenerative Medicine scientists have fine-tuned their delivery system to deliver a DNA editing tool to alter DNA sequences and modify gene function. The improved "hit and run" system works faster and is more efficient.

"With this new method we are able to package together in one lentiviral capsid both essential components (Cas9 protein and guide RNA) for the CRISPR mediated gene editing," said Baisong Lu, Ph.D, assistant professor of regenerative medicine at WFIRM and one of the lead authors of the paper. "Previously, the two components had to be delivered separately which was not as convenient."

CRISPR (clustered regularly interspaced short palindromic repeats) technology is used to alter DNA sequences and modify gene function. CRISPR/Cas9 is an enzyme that is used like a pair of scissors to cut two strands of DNA at a specific location to add, remove or repair bits of DNA. But CRISPR/Cas9 is not 100 percent accurate and could potentially cut unexpected locations, causing unwanted results.

Now the WFIRM team can package the two components—Cas9 protein (the enzyme) and the guide RNA—as ribonucleoproteins inside the lentiviral capsid, creating a lentiviral capsid-based bionanoparticle system for delivering CRISPR/Cas9. Lentiviral vector is a widely used gene delivery vehicle in research labs and is already widely used for delivering the CRISPR/Cas9 machinery for efficient genome editing. However, using lentiviral vector to deliver CRISPR/Cas9 will result in long-term expression of the endonuclease, which is undesired for safety reasons. The new system offers the delivery efficiency of conventional lentiviral vectors, but enables transient Cas9 expression. Thus, the new CRISPR/Cas9 is more efficient and safer.

The research has led the team to hypothesize that a "similar strategy should be translatable to other editor proteins for gene disruption," said Anthony Atala, M.D., director of WFIRM and a co-author of the paper. "We may be able to utilize this to package and deliver other RNPs into , which has been difficult to achieve so far."

The research is part of an ongoing effort to improve in vivo gene editing efficiency which will be useful in research and clinical applications by improving safety and avoiding possible immune responses.

The WFIRM team published its findings in the journal Nucleic Acids Research.

Explore further

Scientists sharpen their molecular scissors and expand the gene editing toolbox

More information: Pin Lyu et al, Delivering Cas9/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for efficient 'hit-and-run' genome editing, Nucleic Acids Research (2019). DOI: 10.1093/nar/gkz605
Journal information: Nucleic Acids Research

Citation: Scientists sharpen gene editing tool (2019, September 13) retrieved 20 October 2019 from
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Sep 13, 2019
Why bother? Let Nature take it course as it has been doing for millions of years.

Sep 13, 2019
Why bother? Let Nature take it course as it has been doing for millions of years.
Nature produced you. We have to be able to fix those kinds of mistakes.

Hey pussytard why do you follow yourself around with a sockpuppet uprating yourself? Its tacky. And greasy. And has no effect on your -2000 deficit.

And anyone who reads your garbage posts knows that's what you're doing. So why bother?

Pussytard, as in "Pirouette, pussycat_eyes, russkiye, obama_socks, RitchieGuy01, racistblackguy, jewsrule, Surveillance_Egg_Unit" your crackpot menagerie. Flea circus. Idiot parade. Something to do with animals.

Sep 14, 2019
Some people did something before the Civil War but conditions today would be determined more by genetic racial differences than distant historical events. Racial genetic differences in intelligence would create differences in behavior that determine success in high IQ created civilization more than surface appearances. If a minority had a standard deviation higher IQ than Caucasians or Asians their behavior would be different and they would be admired. Genetic editing could make both surface appearances and intelligence choices rather than random genetic combinations. The reparations of the civilization of higher intelligence is to fix the errors of nature so that everyone can handle the higher levels of abstraction required by advanced civilization. Racism, like cutting off a limb infected by gangrene, attempts to save a civilization under attack. Gene editing is the better solution.

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