Science Weekly The extraordinary promise of gene editing
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May 22, 2025 A groundbreaking gene-editing therapy has successfully treated a baby with a severe genetic disorder, showcasing a remarkable leap in medical science. Experts discuss innovative techniques, like base and prime editing, that promise to correct DNA mutations linked to various diseases. However, barriers to accessibility remain a concern, highlighting the need for collaboration among scientists, regulators, and funders. The conversation also touches on the alarming decline in federal funding for crucial scientific research and the need for public support.
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KJ's Rare Genetic Treatment
- KJ was born premature with a rare genetic disorder causing toxic ammonia build-up.
- He received a world-first gene-editing treatment targeting his specific mutations within six months.
Base Editing Explains Gene Correction
- Base editing acts like a DNA rewriting pencil, changing one letter in DNA to correct mutations.
- This technique enables precise fixes to genetic diseases by editing DNA at a single-letter level.
KJ's Treatment Progress
- KJ's bespoke base editing treatment was infused three times and shows signs of working.
- The goal is a one-time correction that persists lifelong, though long-term monitoring is needed.