遗传性疾病

A new study using AI-powered sequencing reveals that DNA wrapped around nucleosomes isn't inaccessible, but exists in 14 distinct states of distortion that control gene activity. 四月 29, 2026

Regeneron’s Otarmeni marks the first neurosensory gene therapy to reach the market, yet its rapid path through the FDA raises questions about long-term efficacy and the high cos... 四月 24, 2026

Six-year-old Saffie Sandford’s vision restoration highlights the critical developmental window for gene therapy and the limits of ‘miracle’ narratives. 四月 23, 2026

A single injection of AAV gene therapy delivered a working OTOF gene and improved hearing in ten patients within weeks, researchers report. The results are striking for one rare... 四月 05, 2026

A young Australian woman with a rare Cowden‑syndrome variant describes needing 'virtually every test, every six months, for life'. This article explains what a hereditary cancer... 二月 16, 2026

UBC researchers report a topical CRISPR treatment that repairs disease-causing mutations in human skin models using laser-assisted lipid nanoparticles, a potential platform for ... 一月 27, 2026

A 24-year-old man from Norfolk who developed aggressive frontotemporal dementia donated his brain to Addenbrooke’s Hospital to help researchers study early-onset disease and its... 一月 13, 2026

An investigation this week found sperm from an anonymous donor carrying a TP53 mutation linked to Li‑Fraumeni syndrome was used to conceive at least 197 children across Europe, ... 十二月 11, 2025

Researchers report that loss-of-function variants in the GRIN2A gene can directly produce early-onset psychiatric illness, challenging the idea that major mental disorders are a... 十二月 05, 2025

A new gene‑editing platform called PERT uses engineered suppressor tRNA to bypass premature stop signals in DNA, rescuing protein production in cells and mice and offering a pot... 十二月 05, 2025

A Mammoth Biosciences executive told the Dubai Future Forum that gene editing could treat every genetic disease within ten years. Here’s what the science, recent trials and majo... 十一月 19, 2025