Gene Editing on Demand for Genetic Disease

Gene therapy, put simply, involves introducing, removing, or correcting a gene inside cells to cure diseases. And medical research is finding more ways to apply it. 

The newest method treated a baby dying from toxic buildup of ammonia in his brain because his body couldn’t effectively process protein.  Doctors created the treatment for him, based on his DNA sequence. This personalized gene therapy took decades of research with a big breakthrough in the 1970s when scientists learned how to cut and paste DNA segments.  Two decades later, they inserted a working copy of a gene into a girl’s white blood cells to treat her immune disorder. Human gene therapy had arrived.  

Today, we’ve graduated to the CRISPR-Cas9 gene editor, bringing this game-changing tool out of the lab and into hospitals where it’s treating diseases with single-gene mutations such as cystic fibrosis.  In this baby’s case, lipid nanoparticles carried the “correcting” DNA and a gene editor to target his liver cells and repair his CPS-one gene.  

Billions of these nanoparticles were infused into his blood over three treatments and during that time, his body began tolerating increasing levels of protein with few side effects.  

It took just six months to design this treatment, and “CRISPR on demand” is a platform that could now target other rare genetic diseases. The downsides? It can cost millions and needs long-term studies to make sure there are no unintended effects. Still, gene editing therapy is a glimpse of a future with custom-built care.