Gene therapy articles from across Nature Portfolio Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease.

PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US.

Genetics is the branch of science concerned with genes, heredity, and variation in living organisms. It seeks to understand the process of trait inheritance from parents to offspring, including ...