India White รีทวีตแล้ว

This is INCREDIBLE.
KJ (now 9.5 months old) had a rare monogenic disorder, a mutation of a single gene.
We can now create custom gene-editing therapies tailored to a patient's exact mutation. A lipid bubble delivers instructions and CRISPR, which crawls along the DNA to find and fix the error.
KJ’s treatment was personalized so CRISPR could target his specific mutation. The same method could be reused for others, just swap out the guide for the other genes.
There are 30 million Americans who have rare diseases. This is potentially one of the most transformational technologies in healthcare.
It could also help with more common conditions like sickle cell, cystic fibrosis, Huntington’s, and muscular dystrophy.
The doctor said this “is a triumph for the American peoples’ investment in biomedical research"
The breakthrough was built on decades of U.S.-funded science, from bacterial immunity to CRISPR, genome sequencing, and rare disease research.
“I don’t think this could have happened in any country other than the US" said Dr. Urnov🇺🇸🇺🇸🇺🇸

The New York Times@nytimes
Breaking News: A baby with a rare disorder made medical history by receiving the first custom gene-editing treatment. The technique used has the potential to help people with thousands of other uncommon genetic diseases. nyti.ms/4j3xGs8
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