Six-Month-Old KJ Muldoon Becomes First Baby Treated with Personalized CRISPR Gene Therapy
KJ Muldoon, a baby boy born with a rare genetic disorder, has been successfully treated with CRISPR gene therapy, marking a significant breakthrough in personalized medicine.
In World First, Baby Receives Personalized CRISPR Gene-Editing Treatment
Scientific American
Custom genetic treatment saved infant’s life, study says
World News Group
This baby boy was treated with the first personalized gene-editing drug
MIT Technology Review
Summary
Researchers from Children’s Hospital of Philadelphia have used personalized CRISPR gene-editing therapy to treat KJ Muldoon, a six-month-old baby boy diagnosed with a severe genetic metabolic disease. This unique therapy corrects a specific mutation impeding the production of an essential liver enzyme, which previously posed life-threatening risks due to ammonia buildup in his blood. KJ's health has drastically improved following the treatment, allowing him to consume more protein. His case, presented at a major medical conference, represents not only a potential template for treating similar disorders but also reflects the rapid advancements in personalized medicine and regulatory processes.
Articles (14)
- The use of CRISPR technology represents a significant evolutionary step in medicine, particularly in providing options for patients with rare genetic disorders who would otherwise face dire consequences, as shown in KJ's promising but cautious treatment outcomes.
- Government involvement and funding in biomedical research are crucial, exemplified by the government support that enabled a rapid response to developing KJ's treatment, reflecting the importance of public investment in healthcare advancements.
- Ethical considerations must remain a priority as personalized gene therapies advance, especially as the long-term effectiveness of treatments like KJ's is still being evaluated; ensuring access to these therapies for broader populations remains a pressing concern.
Scientific American·16d· "…It’s an ambitious approach that researchers hope will inspire others to harness CRISPR to treat ultra-rare genetic diseases."
TIME Magazine·16d· "…While the therapy was created for him, the team is hopeful that the process can be made more universal and applied to other genetic mutations, for which they can plug in the appropriate genetic change to correct a disease."
Boston Globe·17d· "…KJ's treatment — which built on decades of federally funded research — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing."
The Guardian·17d· "…The promise of gene therapy that we’ve heard about for decades is coming to fruition, and it’s going to utterly transform the way we approach medicine."
Wired·17d· "…The case is detailed today in a study published in The New England Journal of Medicine and was presented at the American Society of Gene & Cell Therapy annual meeting in New Orleans."
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- 16d