Therapeutic Potential of CRISPR–Cas3 Genome-Editing System for Transthyretin Amyloidosis (IMAGE)

The Institute of Medical Science, The University of Tokyo

Therapeutic Potential of CRISPR–Cas3 Genome-Editing System for Transthyretin Amyloidosis

Caption

In a new study, the researchers have shown that the CRISPR–Cas3 system can induce reliable, extensive deletions of the transthyretin (TTR) gene and reduce production of TTR protein without any unintended DNA cuts. The results highlight its potential as an efficient genome-editing system that can be used to cure transthyretin amyloidosis (ATTR), a progressive disorder involving amyloid deposits of misfolded TTR proteins and other genetic disorders.

Credit

National Institutes of Health (NIH) via Creative Commons Search Repository Image source link: https://openverse.org/image/ce250f8d-cc56-44bd-8b52-61efc4f23217?q=gene+editing&p=2

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