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Could CRISPR-Cas3 present itself as a potential therapeutic approach for genetic disorders like transthyretin amyloidosis (ATTR)?
Researchers from the Institute of Medical Science, The University of Tokyoexternal link, opens in a new tab (IMSUT; Tokyo, Japan) have explored the use of CRISPR-Cas3 as a therapeutic strategy for treating genetic disorders such as ATTR. The findings of the study highlight the efficiency of CRISPR-Cas3, addressing some of the limitations associated with the widely known CRISPR-Cas9 system.
The development of various gene-editing techniques has enabled precise modifications to DNA, allowing for the correction of mutations or the removal of harmful genetic sequences.
“Genome editing holds the unique potential to correct the inherited disease-associated genetic abnormalities,” commented Tomoji Mashimo who co-led the study.
Among these gene-editing tools, CRISPR-Cas9 has gained significant traction for its potential clinical applications; and despite its promise, this approach has certain limitations, such as the risk of off-target DNA editing – unintended cuts in the genome, which can lead to unwanted mutations.