A Baby Received a Custom Crispr Treatment in Record Time
1 min read
Summary
KJ Muldoon was born with a fatal genetic disorder, CPS1 deficiency, which causes excess ammonia to build up in the blood and can lead to organ and brain damage.
Six months after his birth, he received a pioneering Crispr-based medicine, designed and produced in collaboration with the FDA in just a few months.
After three doses of the drug, Muldoon has avoided the usual treatment options of a highly restrictive diet and liver transplant, and has made “tremendous strides,” according to his father.
The case could provide a model for creating custom gene-editing therapies for patients with rare diseases who have limited or no treatment options.
Crispr offers the potential for genetic editing to cure diseases, but delivery of the medicine to the right parts of the body, and ensuring it is safe, are barriers to more widespread use.
