When Xingxu Huang began thinking about correcting disease-causing mutations in the human genome, his attention turned naturally to CRISPR–Cas9. But it quickly became clear that the popular gene-editing tool wasn’t ideal for the majority of human disease mutations, which result from errors in single DNA nucleotides known as point mutations. More than 31,000 such mutations in the human genome are known to be associated with human genetic diseases. But CRISPR is not particularly efficient at correcting them.
Then Huang learnt about base editors, a new class of genome-modifying proteins that excel at single-site mutations.
Read this blog post in full on the Nature Careers website – https://www.nature.com/articles/d41586-019-03536-x