CRISPR
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The era of genomic medicine has begun. A line of powerful tools has been developed to cure genetic diseases. There is a last-mile problem because most of the tools are too big to be used in popular genome therapy.

Cornell scientists provide an explanation for how this problem is solved by nature, using atomic precision to define how a transposon-derived system edits DNA. There are mobile genetic elements inside the bacterium. The IscB is less than half the size of the other genes in the transposon family and is capable of DNA editing. IscB would solve the size problem.

The paper was published in Science on May 26.

The researchers used a high resolution microscope to see the IscB-ωRNA molecule from a transposon system. They were able to take snapshots of the system in different states.

Next-generation fancy applications require the gene editor to be fused with other enzymes and activities, and most Cas9s are already too big for viral delivery. We are facing a traffic jam at the delivery end, if we can package the Cas9s into viral vectors that have been used for decades in the gene therapy field.

A guide to recognize a sequence of DNA is provided by the CRISPR-Cas9 systems. The data gathered by the Cornell team shows that it is possible to do surgery at the DNA level to fix genetic diseases.

The structure of the molecule and how it performs the chemical reactions are the focus of the study.

It is believed that transposons were the beginning of the evolution of CRISPR systems. Barbara McClintock discovered them.

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The researchers believe they will be able to shrink IscB even more. They hope to make future versions of this genome editor even smaller and more useful because they have already removed 55 of the 55 amino acids.

There is still a lot of mystery about the function of the companion guide RNA, said co-first author Chunyi Hu. What other roles is thisRNA playing?

While the IscB-ωRNA is active in test tubes, it was not as efficient at altering human cells. The next step in their research will be to use the structure of the molecule to see if there is a cause for the low activity in human cells.

More information: Gabriel Schuler et al, Structural basis for RNA-guided DNA cleavage by IscB-ωRNA and mechanistic comparison with Cas9, Science (2022). DOI: 10.1126/science.abq7220 Journal information: Science Citation: Discovery offers starting point for better gene-editing tools (2022, May 26) retrieved 26 May 2022 from https://phys.org/news/2022-05-discovery-gene-editing-tools.html This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.