Recent research conducted by a team at Montana State University has demonstrated how RNA, closely related to DNA, can be edited using CRISPR technology. This breakthrough offers a promising avenue for treating various genetic disorders. Postdoctoral researchers Artem Nemudryi and Anna Nemudraia, alongside Professor Blake Wiedenheft, spearheaded the study, titled “Repair of CRISPR-guided RNA breaks enables site-specific RNA excision in human cells,” published in the journal Science [1]. Their work represents a significant advancement in utilizing CRISPR for genetic engineering, particularly in RNA manipulation.

Figure 1. Anna Nemudraia, left, and Artem Nemudryi. (Credit: Adrian Sanchez-Gonzalez)

Figure 1 shows Anna Nemudraia, left, and Artem Nemudryi are lead authors on a new paper exploring the use of CRISPR technology to edit RNA genetic material.

While CRISPR has been extensively used for DNA editing, the team's innovation lies in applying similar techniques to RNA, a process never before achieved. Previous experiments had shown the potential of CRISPR in editing viral RNA in controlled environments, but the team aimed to explore its efficacy within living human cells [2].

Programming type-III CRISPR proteins, the researchers successfully targeted RNA containing a cystic fibrosis-causing mutation, effectively restoring cellular function. Surprisingly, upon sequencing the RNA, they discovered that the cell had naturally repaired the RNA, removing the mutation [1].

This discovery challenges the conventional understanding of RNA's transient nature within cells, suggesting that RNA repair might be a fundamental biological process. Mentorship from Wiedenheft, who has guided Nemudryi and Nemudraia throughout their postdoctoral tenure, has been instrumental in their groundbreaking contributions. The potential of RNA editing in treating genetic diseases is immense, offering reversible edits with lower risk compared to DNA manipulation.

The team's success in removing a cystic fibrosis-causing mutation is just the beginning. They anticipate further exploration into addressing a myriad of genetic mutations using this RNA editing technology. As they transition to faculty positions at the University of Florida, they remain committed to pushing the boundaries of science, emboldened by Wiedenheft's encouragement to explore uncharted territories in research.

Source: Montana State University

References:

1. https://www.eurekalert.org/news-releases/1042968
2. https://www.news-medical.net/news/20240429/RNA-editing-using-CRISPRs-shows-promise-for-genetic-disease-treatment.aspx

Cite this article:

Hana M (2024), CRISPR Editing Unlocks RNA Manipulation Potential, AnaTechmaz, pp. 248