Gene editing tools have been on the rise lately. In a previous post on BioDiaries, you can see how CRISPR (gene editing) is expected to boom in 2025. A week after the New Year post, let us explore an innovation that not just helps us edit genes, but several other functions. Let us dive deep and see how this innovation will benefit us.
How do gene editing tools help treat genetic diseases?
Genetic diseases are heritable and occur due to defects in genes. To treat such conditions, we require flexible gene editing tools like CRISPR to edit genomes (DNA) and transcriptomes (RNA). In addition, gene editing tools are a primary requirement for establishing treatments, genetic screening, and metabolic engineering.
We need a separate set of tools to regulate the gene expression. Tools like CRISPR/Cas combined with transcriptional activators have been in use lately. They help activate, and silence genes or edit them. However, CRISPR/cas relies on homology-directed repair involving double-stranded breaks (DSBs). Inefficient in many types of cells that are important for medical treatments. Moreover, DSBs can cause unintended genetic changes and even cell damage, raising safety concerns for therapies.
Alternative tools like base editors are currently in use. They can carry out single-letter changes in the DNA without causing double-stranded breaks. Although better than DSBs, base editors can only edit point mutations and cannot change a short to medium-length sequence. Moreover, they cannot edit and activate genes at the same time. This brought scientists to explore alternative and safer tools to edit and regulate gene expression.
Minimal versatile genetic perturbation technology (mvGPT)
With this versatile and novel tool, we can edit, activate, and repress human genes with greater accuracy and efficiency. The main components of this tool include- a prime editor, a fusion activator (comprising of cas9 and reverse transcriptase), and a guide RNA (PEG RNA) which generates RNA tailor-made to different genetic changes without causing double-stranded breaks. mvGPT combines the prime editor with an editing guide RNA and a nicking guide RNA to edit genomes.
To activate gene expression, the prime editor is combined with a synergistic activation mediator system.
To silence gene expression, they employ short hairpin RNA (shRNA)
To understand mvGPT, we need to understand DAP.
This is because this versatile gene editing tool was based on DAP. It organizes RNA molecules in a compact format to simplify gene editing tasks. Using a short human RNA (hCtRNA) to act as a promoter and spacer, ensures RNA components are expressed efficiently without needing multiple complex promoters. The array produces different RNA tools for various functions including gene editing, gene activation, and gene repair.
Just as this paper shows, mvGPT has numerous real-world applications and works with many delivery methods including AVV and lentivirus.
Overall, mvGPT is a powerful, all-in-one tool that edits, activates, or silences genes efficiently and precisely. It’s compact, versatile, and works with multiple delivery methods, making it a promising solution for treating genetic diseases.
What do you think about this versatile tool? Let me know in the comments.
Thanks for sharing. Great read.