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Enhanced human genome editing method could amplify the capability and accuracy in manipulating genetic code

Scientists Discovered a Novel Genome Editing Method — CRISPR-Cpf1 — That Might Bolster Genetic Modification's Efficiency and Precision. This new technique appears to streamline the process of DNA editing compared to currently employed methods like CRISPR-Cas9.

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Scientists have discovered a novel method for modifying the human genome, promising enhanced...
Scientists have discovered a novel method for modifying the human genome, promising enhanced efficiency and precision in genetic editing. The new tool, CRISPR-Cpf1, provides a more straightforward means of altering DNA compared to current techniques such as CRISPR-Cas9.

Enhanced human genome editing method could amplify the capability and accuracy in manipulating genetic code

Scientists, led by the researcher who initially pioneered the use of CRISPR-Cas9 for gene editing in mammals, have discovered a new CRISPR system with potential for simpler and more precise genome engineering. The findings, published in Cell, describe the unique characteristics of this novel system, known as Cpf1, and demonstrate its ability to edit human DNA.

The study, carried out by a team that includes Feng Zhang from the Broad Institute of MIT and Harvard, and the McGovern Institute for Brain Research at MIT, along with co-authors from the National Institutes of Health, the Broad Institute, the MIT Department of Biology, and Wageningen University, reveals the unexpected features of Cpf1 and its potential for human genome editing.

"This could dramatically advance genetic engineering," explains Eric Lander, director of the Broad Institute. He adds that the Cpf1 system has powerful and remarkable features that make it a new generation of genome editing technology.

The research team searched through hundreds of CRISPR systems in various bacterial species, seeking enzymes with beneficial properties applicable to human cells. They identified the Cpf1 enzymes from the bacterial species Acidaminococcus and Lachnospiraceae and demonstrated their effectiveness in targeting genomic loci in human cells.

"We were delighted to discover completely different CRISPR enzymes that can be harnessed for genome editing research and human health," says Zhang, the W.M. Keck Assistant Professor in Biomedical Engineering at MIT.

Compared to the previously known Cas9, Cpf1 has several significant differences. In its natural form, Cas9 requires two small RNAs for DNA cutting activity, whereas Cpf1 only needs a single RNA, simplifying its design and potentially making it easier to deliver into cells and tissues.

Moreover, Cpf1 cuts DNA in a different manner than Cas9, leaving staggered or "sticky" double-stranded breaks. This is expected to facilitate more precise insertion, allowing researchers to integrate a piece of DNA more efficiently and accurately. Additionally, Cpf1 cuts far away from the recognition site, which could enable multiple opportunities for correct editing if the targeted gene becomes mutated at the cut site.

The Cpf1 system also provides new flexibility in choosing target sites, as it recognizes a different type of PAM sequence than Cas9, potentially expanding the range of potential targets, including in human and malaria parasite genomes.

Levi Garraway, an investigator at the Broad Institute, notes that the properties of Cpf1 and more precise editing have potential applications in cancer research.

To ensure the widespread availability of the Cpf1 system, Zhang, the Broad Institute, and MIT plan to share it freely with academic researchers via the Addgene plasmid-sharing website. They will also offer licenses for commercial use, with a focus on rapid and safe development for therapeutic applications.

Zhang expresses the team's commitment to making the CRISPR-Cpf1 technology readily available, aiming to accelerate research and develop new therapeutic applications. He hints at future potential for other enzymes to contribute to further advances in genome editing.

  1. The newly discovered CRISPR system, Cpf1, shows potential for simpler and more precise genome engineering in human cells, a breakthrough reported in Cell.
  2. According to Eric Lander, director of the Broad Institute, the Cpf1 system represents a new generation of genome editing technology with powerful and remarkable features.
  3. The research team, including Feng Zhang and colleagues from MIT, the National Institutes of Health, and Wageningen University, identified Cpf1 enzymes from bacterial species Acidaminococcus and Lachnospiraceae, and demonstrated their effectiveness in human cells.
  4. Compared to Cas9, Cpf1 has several significant differences, such as requiring only a single RNA for DNA cutting activity, leaving staggered double-stranded breaks, and cutting DNA far away from the recognition site, potentially enabling multiple opportunities for correct editing.
  5. Levi Garraway from the Broad Institute suggests that the properties of Cpf1 and more precise editing have potential applications in cancer research.
  6. To promote the widespread use of the Cpf1 system, Zhang, the Broad Institute, and MIT plan to share it freely with academic researchers via the Addgene plasmid-sharing website and offer licenses for commercial use, with a focus on rapid and safe development for therapeutic applications.

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