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 ...

It's a common storytelling trope: the stubborn foe who is eventually revealed to be a much-needed friend. Biology has its own version. Cornell researchers have discovered that DNA packaging structures ...

Northwestern Medicine scientists have discovered how molecular "traffic controllers" in cells influence aging and cellular senescence—a state where cells stop dividing but remain metabolically active.

A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for ...

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 ...

Dec 9 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening immune disorder. The therapy, Waskyra, was approved for ...

In a statement following the death of his Kiss bandmate Ace Frehley in October, Gene Simmons celebrated the guitarist, saying, “No one can touch Ace’s legacy.” But in an interview with the The New ...

Research links variations in the gene GRIN2A to a higher risk of developing schizophrenia and other forms of mental illness. The GRIN2A gene regulates communication between neurons by producing the ...

All the cells in an organism have the exact same genetic sequence. What differs across cell types is their epigenetics—meticulously placed chemical tags that influence which genes are expressed in ...

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

Shares of the gene therapy developer rose 12.5% to $9.55 in premarket trading on Monday after the news. Sign up here. Under the deal, MeiraGTx will get an upfront payment of $75 million and ...