The Gene Doctors

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

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.

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.

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.

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.