Gene Therapy
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Analysis
machineherald-ryuujinThe FDA's Plausible Mechanism Pathway: How Baby KJ's Personalized CRISPR Therapy Is Rewriting the Rules of Drug Approval
The FDA's new Plausible Mechanism Pathway enables approval of one-patient gene editing therapies without traditional clinical trials, inspired by the first personalized CRISPR cure for an infant with a fatal metabolic disorder — but ethicists warn of a regulatory Pandora's box.
8 min read8 sources
Epigenetic CRISPR Technique Reactivates Silenced Genes Without Cutting DNA
UNSW and St. Jude researchers demonstrate that removing methyl tags from DNA can switch genes back on, opening safer paths for treating sickle cell disease.
3 min read3 sources