16 April 2025 - Baby treated with first-ever personalized gene editing treatment
CRISPR is used in landmark treatment to correct genetic misspelling of a single patient
Jason Mast (STAT News, 15/05/2025)
For the first time, scientists say they have reached into the genome of a severely ill child and rewritten the unique misspelling in his DNA. The results, published in the New England Journal of Medicine on Thursday, are a landmark in the 50-year quest to read and repair the code of life. The boy, a now 9.5-month-old named KJ, was diagnosed days after birth with an ultra-rare disease that impairs his liver’s ability to process ammonia, which can build up and cause permanent brain damage or death.
A promising genetic treatment tailor-made for a baby born with a rare disorder
Rob Stein (NPR, 15/05/2025)
For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby received three infusions containing billions of microscopic gene-editors that homed in on a mutation in his liver and appear to have corrected his defect. Doctors need to follow the boy longer to determine how well the treatment is working. But so far the bespoke therapy appears to have at least partially reversed his condition, reducing his risk of suffering brain damage and possibly even death.
A Pennsylvania baby is the first to successfully receive personalized gene therapy
The Associated Press (NBC News, 15/05/2025)
A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, saying he’s among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his genetic code that kills half of affected infants. Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can someday help the millions left behind even as genetic medicine has advanced because their conditions are so rare.
Beyond CRISPR: Scientists Develop New Tool for Cutting DNA
INRS (SciTechDaily, 09/04/2025)
A few years ago, the introduction of CRISPR technology marked a significant breakthrough in the scientific community. Derived from a component of the bacterial immune system, CRISPR enables precise cuts in double-stranded DNA, allowing scientists to modify specific genes in plants, animals, and humans. This precision has made CRISPR a leading tool in the development of treatments for both inherited and acquired diseases.
Crystal structure of a CRISPR. Wikipedia, CC BY-SA 4.0.
