Medical Discovery News
Science permeates everyday life. Yet the understanding of advances in biomedical science is limited at best. Few people make the connection that biomedical science is medicine and that biomedical scientists are working today for the medicine of tomorrow. Our weekly five-hundred-word newspaper column (http://www.illuminascicom.com/) and two-minute radio show provide insights into a broad range of biomedical science topics. Medical Discovery News is dedicated to explaining discoveries in biomedical research and their promise for the future of medicine. Each release is designed to stimulate listeners to think, question and appreciate how science affects their health as well as that of the rest of the world. We also delve into significant biomedical discoveries and portray how science (or the lack of it) has impacted health throughout history.
Medical Discovery News
Gene Editing on Demand for Genetic Disease
990 Gene Editing on Demand for Genetic Disease
Welcome to Medical Discovery News. I’m Dr Norbert Herzog.
And I’m Dr. David Niesel
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 nineteen seventies 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.
We are Drs. David Niesel and Norbert Herzog, at UTMB and Quinnipiac University, where biomedical discoveries shape the future of medicine. For much more and our disclaimer go to medicaldiscoverynews.com or listen to our podcast on your favorite podcast service.
