David Liu's lab develops next-gen base editors, small enough to fit into single AAV
Researchers use adeno-associated viruses (AAVs) as vehicles to deliver CRISPR/Cas systems, correct gene copies and DNA and RNA sequences into human cells as treatment modalities. But the size of the viruses themselves has been a limiting factor for cargo that can be stuffed into it. For instance, gene-editing technologies such as CRISPR/Cas and base editors must be divided into two adenoviruses.
Now, for the first time, scientists have engineered a way to shrink base editors, allowing them to be loaded into a single virus particle. David Liu, from MIT and Harvard’s Broad Institute, led the research and published a paper Thursday in Nature Biomedical Engineering.
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