The first gene therapy treatment in the United States was approved recently by the Food and Drug Administration, heralding a new era in medicine that is coming faster than most realize — and that perhaps few can afford

Quick Read

The disease may be among the first to be treated with the novel gene-editing tool

Quick Read

How an unusual medical case in the 1990s provided a clue for how to treat a fatal muscle disease

Quick Read

GlaxoSmithKline says it has treated a child with Strimvelis, its gene therapy for immune deficiency

Quick Read

Families of patients are starting advocacy groups, raising money for research, and founding biotech companies to advance cures for rare diseases

Quick Read

The first-in-the-world case is detailed in Thursday’s New England Journal of Medicine

Quick Read

So far, gene therapy has only treated rare disorders. Now, for the first time, it has been used to treat a boy with sickle cell disease, a common genetic disease

Quick Read

A one-off injection could one day lower your cholesterol levels for the rest of your life. People born with natural mutations that disable a specific gene have a lower risk of heart disease, with no apparent side effects. Now a single injection has successfully disabled this same gene in animal tests for the first time

Quick Read