Genetics

Clinical evidence, research, and medical intelligence in Genetics.

DMD base editing with liver-sparing AAV improves muscle disease in mice

A compact liver-sparing myotropic AAV delivered adenine base editing in humanized DMD mice, restoring reading frame and improving muscular dystrophy.

Ancient DNA from bones and cave sediments reveals long-distance connections, regional continuity, and Ice Age migration across Paleolithic…

Researchers used a high-throughput minigene assay to map pseudoexon-activating deep intronic variants in NF1 and benchmark splicing predictors.

The NIH Genetic Testing Registry centralizes global genetic test information, including purpose, validity, methods, and laboratory details.

Patient-led rare disease drug development can speed therapies, but it also creates ethical tensions with academics, industry, and…

FDA draft guidance would ease approval rules for some individualized gene therapies, with a plausible mechanism pathway for…

A protein-folding informed Bayesian model improved hearing-loss variant classification and upgraded genetic diagnoses for 12 probands in a…

NIH’s Genetic Testing Registry consolidates global genetic test details, including purpose, validity, methods, and lab credentials, into one…

Cell Genomics marks five years with reflections from Chinese authors on publishing, community, and the direction of genomics…

A nonprofit is reviving Bloomsbury Genetic Therapies’ lead gene therapy after the U.K. startup closed after dosing one…

Genetics

Clinical evidence, research, and medical intelligence in Genetics.

In Brief The 5-minute rounds