Researchers found that paired pathogenic variants can restore protein function rather than make it worse. This phenomenon ...

Euan Ashley's lab explores the intricate interactions of gene variants. Tiny "typos," or genetic mutations, can sneak into segments of DNA. Many of these are harmless, but some can cause health ...

In 2018, a nervous-looking He Jiankui took the stage at a scientific conference in Hong Kong. A hush settled over the packed auditorium as the soft-spoken Chinese scientist adjusted his microphone and ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

This year marked a pivotal moment in the quest to treat Huntington’s disease, a rare but devastating form of dementia. Scientists found that an experimental gene therapy slowed the condition’s ...

KJ Muldoon is walking at home ahead of Christmas after being hospitalized last holiday due to his rare genetic disorder, CPS1 deficiency Getty KJ Muldoon, the first infant to undergo gene-editing ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the first therapy of its kind for the rare disease and making Italy’s Fondazione ...

Andrew Joseph covers health, medicine, and the biopharma industry in Europe. You can reach Andrew on Signal at drewqjoseph.71. The Food and Drug Administration this week granted approval to a new gene ...

In a breakthrough study, scientists have discovered that a variant in one gene, GRIN2A, can directly cause mental illness – something previously believed to be the result of several mutations working ...

Scientists have discovered that a single gene, GRIN2A, can directly cause mental illness—something previously thought to stem only from many genes acting together. People with certain variants of this ...

With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for medication, according to a new pilot study published Saturday in the New ...

Late last year, dozens of researchers spanning thousands of miles banded together in a race to save one baby boy’s life. The result was a world first: a cutting-edge, gene-editing therapy fashioned ...