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A recent study in Genome biology shows how CRISPR-SKIP, a new gene editing method, offers tremendous potential for many biomedical applications.
What if doctors could treat previously incurable genetic diseases caused by errors or gene mutations? Thanks to new research done by American scientists from the University of Illinois, we are on the verge of realizing this reality. Posted in Genome biology, their work is based on CRISPR-Cas9, a revolutionary genome editing system.
In general, body cells "read" DNA to produce the proteins needed for different biological functions. Scientists can change the way in which DNA is read using CRISPR gene modification technology. CRISPR-Cas9 is often used to cut out specific areas of DNA and repair faulty genes. In this study, the researchers modified the existing technology to create CRISPR-SKIP. Instead of breaking DNA to eliminate faulty genes, CRISPR-SKIP modifies a single base of the targeted DNA sequence, causing the cell to ignore reading this section of the DNA.
According to the authors of the study, CRISPR-SKIP can permanently eliminate the defective DNA sections, thus making it possible to sustainably treat certain genetic diseases in a single treatment. They have successfully tested their technique on mouse and human cell lines. The scientists aim to test the method on living organisms in the future.
CRISPR-SKIP can potentially help treat many diseases such as cancer, rheumatoid arthritis, Huntington's disease and Duchenne muscular dystrophy, to name a few. As the method only requires the editing of a single database, it is simple, accurate and adaptable to a variety of cell types and applications.
Written by Cindi A. Hoover, Ph.D.
The references:
- Gapinske et al. CRISPR-SKIP: Programmable gene splicing with single-base editors. 2018. Genome biology 19: 107.
- New CRISPR technique that skips portions of genes that can cause disease. https://news.illinois.edu/view/6367/683492
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