Engineering a New Virus to Deliver Gene-Editing Tools | AIChE

Engineering a New Virus to Deliver Gene-Editing Tools

July
2023

A virus that evolved to infect bacterial cells may be the next tool in the arsenal for genetic engineering.

Researchers have discovered a way to tweak bacteriophage T4, a virus that typically infects the bacterium Escherichia coli, to enter human cells and deliver gene-editing tools such as CRISPR-Cas9. The bacteriophage is incapable of infecting human cells and causing disease, but it can deliver DNA, RNA, and other molecules to edit the cell’s genetic instruction manual. The ultimate goal is to use this delivery vehicle to conduct gene therapy, curing diseases such as muscular dystrophy, which results in the progressive weakening of the muscles.

All methods of gene therapy require a vector to deliver the gene-editing tools into the cell. So far, most research has focused on viruses adapted to infect humans, such as adenoviruses and lentiviruses. These viruses can be “defanged” of their disease-causing potential and packed with snips of DNA to repair the genome, but they suffer from size limitations, says Venigalla Rao, a biologist at the Catholic Univ. of America and one of the lead developers of the new bacteriophage tool.

A typical adeno- or lentivirus can hold genetic sequences of only 5,000–8,000 base-pairs long, Rao says, which is much shorter than many complex genes that cause diseases. (Base pairs are the fundamental units of DNA sequences.)

 

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