This baby boy was treated with the first personalized gene-editing drug
1 min read
Summary
Researchers focused their CRISPR editing on a single base in a single gene, CPS1, knowing that a mutation there led to a fatal metabolic condition.
They designed their editing tool to home in on that spot, and flushed out the cells that they’d successfully edited with a coloured chemical.
This is the first use of gene editing to treat an individual case, and while the treatment is unlikely to be used again, it shows the potential of gene editing to save lives.
The real challenge, however, will be in scaling up the process to make it cheaper and more accessible.
Kyle “KJ” Muldoon Jr was suffering from a rare metabolic condition caused by a mutation in the CPS1 gene, which plays a key role in breaking down amino acids.