CRISPR Gene Therapy: Smaller Protein for Viral Delivery
- A new version of a CRISPR protein could revolutionize gene therapy.
- The team, led by Hongjian wang, focused on developing a more effective tool for CRISPR gene-editing systems.
- One strategy involves using non-pathogenic viruses to deliver CRISPR proteins to target cells, modifying DNA sequences to combat diseases. However, the adeno-associated virus (AAV), a popular choice, has...
Researchers have made a significant breakthrough in gene therapy, unveiling enEbCas12a, a novel and compact CRISPR protein from Wuhan University. This innovative protein demonstrates efficient gene editing and holds promise for potential cholesterol treatment, marking a pivotal step forward in medical science. Notably, this smaller protein is ideally suited for virus-based delivery, a crucial advancement. Experiments on mice revealed significantly reduced cholesterol levels after treatment, showcasing enEbCas12a’s therapeutic potential and the impact it could have on future clinical applications.Discover how this could transform approaches to treating diseases, and explore the implications as News Directory 3 reports on the latest in gene editing technology.Discover what’s next for enEbCas12a.
Novel CRISPR protein Shows Promise for Gene Therapy
Updated June 12, 2025
A new version of a CRISPR protein could revolutionize gene therapy. Researchers at Wuhan University in China have engineered a novel CRISPR-associated (Cas) protein, offering efficient gene-editing activity. their findings, focusing on the potential of this protein for cholesterol treatment, were published in PLOS biology.
The team, led by Hongjian wang, focused on developing a more effective tool for CRISPR gene-editing systems. These systems, naturally present in bacteria, are increasingly explored for treating human diseases. CRISPR systems rely on Cas proteins, with Cas9 and Cas12a being the most common.
One strategy involves using non-pathogenic viruses to deliver CRISPR proteins to target cells, modifying DNA sequences to combat diseases. However, the adeno-associated virus (AAV), a popular choice, has limited space. While some Cas9 proteins fit, Cas12a proteins are often too large.
Wang’s team identified a smaller Cas12a variant, EbCas12a, from Erysipelotrichia bacteria. By modifying an amino acid, they enhanced its gene-editing capabilities. The resulting protein,enEbCas12a,demonstrated gene-editing efficiency comparable to other accurate Cas12a proteins when tested on mammalian cells.
The team then demonstrated enEbCas12a’s suitability for AAV-based gene therapy.They modified it to target a cholesterol-related gene, packaged it into a virus, and administered it to mice with high cholesterol.After a month,treated mice showed significantly lower cholesterol levels than untreated mice,highlighting its potential for cholesterol treatment.
The authors add, “The novel compact enEbCas12a, along with its crRNA, can be packaged into an all-in-one AAV system for convenient gene editing in vitro and in vivo with high-fidelity, which can be very beneficial for future clinical applications and more tool developments including all-in-one AAV- based multi-gene editing, base editing, primer editing, etc.”
What’s next
Further research is necessary to determine enEbCas12a’s applicability to human diseases.Though, these results suggest that AAV delivery of Cas12a proteins could become a viable option for future gene therapy applications.