Scientists at the CSIR-Institute of Genomics and Integrative Biology in New Delhi have developed a new genome-editing system that surpasses current CRISPR technology in both precision and efficiency.
The Limitations of Traditional CRISPR
CRISPR, originally a bacterial immune system, was adapted to edit the DNA of complex organisms. However, the traditional CRISPR-Cas9 system sometimes causes "off-target" effects, altering unintended parts of the genome. This issue is particularly pronounced with the SpCas9 enzyme from Streptococcus pyogenes, which, despite improvements in accuracy, often suffers from reduced efficiency.
Advancements with FnCas9
To overcome these challenges, researchers explored the Cas9 enzyme from Francisella novicida, known as FnCas9. Initially, FnCas9 was highly precise but had low efficiency. Scientists led by Debojyoti Chakraborty and Souvik Maiti engineered new versions of FnCas9 to enhance its effectiveness without sacrificing precision. By modifying the amino acids that interact with the target DNA, they improved FnCas9’s binding affinity, making gene editing more effective and flexible.
Improved Performance in Tests
Tests revealed that the enhanced FnCas9 cut DNA more efficiently and could identify single-nucleotide changes in the genome better than the unmodified version. This increased efficiency shows promise for applications in diagnostics and therapeutics.
Practical Applications and Successes
In practical tests, the improved FnCas9 was used to edit human kidney and eye cells. It outperformed traditional CRISPR systems with minimal off-target effects. Researchers also tested its ability to correct a genetic mutation causing Leber congenital amaurosis type 2 (LCA2), a form of inherited blindness. The enhanced FnCas9 successfully corrected the mutation in patient-derived stem cells, leading to normal protein levels in retinal cells.
This advancement offers a more precise tool for genetic editing, potentially improving treatments for genetic disorders. It is especially promising for conditions like LCA2, where traditional methods might be less reliable. Researchers highlight that while precise editing is crucial, effective delivery of the tool into target cells is also essential for successful treatments.
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