The Gene Doctors

Brothers Austin and Max were born with Duchenne Muscular Dystrophy, a genetic disease that prevents the body from making a critical muscle protein. A new medicine that targets the root cause of their disease is giving the boys hope — a treatment that once seemed impossible is now a reality.

Not long after Molly was born, her parents suspected that her vision was impaired — eventually, they discovered she had inherited a genetic mutation that caused her blindness. But recent advancements in genetic therapy have given Molly the chance to see again.

Cystic fibrosis one of the most common fatal genetic diseases in the US, where in patients like Kimberly, a thick coat of mucus in the lungs leads to infections that can destroy airways. The source of the disease is a mutated gene, and the latest treatments target the proteins those mutant genes encode.

See how a revolutionary technology called CRISPR is creating new opportunities for scientists who are developing treatments for genetic diseases.

Every year, a million babies are born worldwide with hereditary diseases. Physicians once had little to offer. But now a new breed of gene doctors is on the case. Devising treatments that target the root causes, they are transforming patients’ lives.