Rationale engineering generates a compact new tool for gene therapy

 


Researchers redesign a compact RNA-guided enzyme from bacteria, making it an efficient editor of human DNA.

Scientists at the McGovern Institute for Brain Research at MIT and the Broad Institute of MIT and Harvard have re-engineered a compact RNA-guided enzyme they found in bacteria into an efficient, programmable editor of human DNA. 

The protein they created, called NovaIscB, can be adapted to make precise changes to the genetic code, modulate the activity of specific genes, or carry out other editing tasks. Because its small size simplifies delivery to cells, NovaIscB’s developers say it is a promising candidate for developing gene therapies to treat or prevent disease.

The study was led by Feng Zhang, the James and Patricia Poitras Professor of Neuroscience at MIT who is also an investigator at the McGovern Institute and the Howard Hughes Medical Institute, and a core member of the Broad Institute. Zhang and his team reported their open-access work this month in the journal Nature Biotechnology.

NovaIscB is derived from a bacterial DNA cutter that belongs to a family of proteins called IscBs, which Zhang’s lab discovered in 2021. IscBs are a type of OMEGA system, the evolutionary ancestors to Cas9, which is part of the bacterial CRISPR system that Zhang and others have developed into powerful genome-editing tools. Like Cas9, IscB enzymes cut DNA at sites specified by an RNA guide. By reprogramming that guide, researchers can redirect the enzymes to target sequences of their choosing.

IscBs had caught the team’s attention not only because they share key features of CRISPR’s DNA-cutting Cas9, but also because they are a third of its size. That would be an advantage for potential gene therapies: compact tools are easier to deliver to cells, and with a small enzyme, researchers would have more flexibility to tinker, potentially adding new functionalities without creating tools that were too bulky for clinical use.

From their initial studies of IscBs, researchers in Zhang’s lab knew that some members of the family could cut DNA targets in human cells. None of the bacterial proteins worked well enough to be deployed therapeutically, however: the team would have to modify an IscB to ensure it could edit targets in human cells efficiently without disturbing the rest of the genome.

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