baby treated for a rare genetic disorder, CPS1 deficiency, with a CRISPR base editing therapy in LNPs. Children’s Hospital of Philadelphia and UPenn researchers. they came up with a brand new gene editing therapy and got FDA approval in 6 months. two IV infusions seem to have fully done the trick!
links 5/16/25: https://roamresearch.com/#/app/srcpublic/page/05-16-2025
https://www.nytimes.com/2025/05/15/health/gene-editing-personalized-rare-disorders.html
baby treated for a rare genetic disorder, CPS1 deficiency, with a CRISPR base editing therapy in LNPs. Children’s Hospital of Philadelphia and UPenn researchers. they came up with a brand new gene editing therapy and got FDA approval in 6 months. two IV infusions seem to have fully done the trick!
https://en.wikipedia.org/wiki/Carbamoyl_phosphate_synthetase_I is a mitochondrial gene involved in the production of urea. without it you get ammonia buildup whenever you consume protein.
https://www.nejm.org/doi/full/10.1056/NEJMoa1910549 pembrolizumab is real good with chemo for stage 2-3 triple-negative breast cancer. either way we’re looking at >50% complete responses.
https://scientific-paper-planner-test.vercel.app/ beta AI app for planning scientific research projects