Adenoviruses used in gene editing experiments have reached their cargo carrying limits and smaller enzymes are crucial to make gene therapy a reality.
Using the new genetic information coded into the nucleus, the cell then continues to function to produce new traits, which can potentially reverse a genetically inherited condition as well.Gene editing using CRISPR-Cas9 has the potential to radically change our world. However, the relatively recent discovery has reached its limitations.
The Cas9 protein is rather large; therefore, fewer enzymes can be packed into the AAVs. This limits the number of enzymes that can be carried into the cell, which has been a roadblock in advancing gene therapy. Researchers have been looking for small alternates and have found one in the Cas12 protein and found that the bacteriumhas the most compact Cas12 enzyme. Dubbed AsCas12f, the Cas12 of the bacterium is only one-third the size of a regular Cas9. The only problem was that it barely showed any gene editing abilities inside human cells.
The engineering team then improved AsCas12f and found that it exhibited 10 times as much editing ability as its unmodified counterpart. However, there exists a possibility that mutations included by the team may not be the most optimal ones. So, the researchers plan to use computational modeling to go through the possible combinations and make an even more powerful Cas12 enzyme.
Experiments on live mice have demonstrated that the engineering AsCas12f, though smaller, is at least as effective as the Cas9 protein. "our next challenge is to actuallyThe research findings were published today in the journal
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Newly engineered CRISPR enzyme for editing DNA could improve patient treatmentA new CRISPR-based gene-editing tool has been developed which could lead to better treatments for patients with genetic disorders. The tool is an enzyme, AsCas12f, which has been modified to offer the same effectiveness but at one-third the size of the Cas9 enzyme commonly used for gene editing. The compact size means that more of it can be packed into carrier viruses and delivered into living cells, making it more efficient.
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