Unveiling a groundbreaking discovery, a team of researchers led by the Feng Zhang at the Broad Institute has discovered a eukaryotic programmable RNA-guided system.

Published in the journal Nature, their study unveils a system centered around the Fanzor protein. The team has demonstrated that Fanzor proteins utilize RNA as a precise guiding mechanism to target DNA. This breakthrough discovery suggested Fanzor bears the potential as a promising therapeutic tool for delivering effective treatments in gene therapies.

Previously, CRISPR/Cas systems were thought to only be associated with prokaryotes, such as bacteria and other single-cell organisms lacking nuclei. Zhang’s paper for the first time demonstrated similar systems existing within eukaryotes. Now, this study confirms the presence of RNA-guided DNA-cutting mechanisms throughout all kingdoms of life.

“CRISPR-based systems have gained tremendous popularity due to their versatility and adaptability in targeting diverse sites within the genome,” enthused Zhang, the corresponding author of the study and a distinguished member of the Broad Institute. He further added, “Our innovative system represents an extraordinary advancement in making precise alterations to human cells, offering an invaluable complement to our existing genome editing toolkit.”

With continuous optimization effort, one should believe that this new system will be listed as an important part of the gene editing toolbox, unlocking even brighter future for the gene-editing based therapies.

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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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