The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

When Demetri Maxim was seven years old, his mother’s kidneys stopped working. She was put on dialysis, which meant that she had to go to the hospital four times a week to have her blood filtered by a ...

A new study has pinpointed how the APOE4 gene variant sabotages the brain’s energy balance, blocking neurons from burning fat for fuel when glucose runs low, a discovery that could reveal new ways to ...

Advanced biotechnology repurposes two bacterial immune systems to correct large stretches of DNA. Human cells that have been edited with the new retron-based gene editing technology. Orange dots mark ...

Biogen has discontinued all gene therapy programs using adeno-associated virus (AAV) capsids, instead shifting resources to modalities that have the highest likelihood of achieving better treatment ...

Hamza is a gaming enthusiast and a Writing Specialist from Pakistan. A firm believer in Keyboard/Mouse supremacy, he will play Tekken with WASD if you let him. He has been writing about games since ...

How can altering DNA help defeat one of the world’s deadliest diseases? This video explores how scientists are using CRISPR and other genetic editing tools to fight cancer - by targeting mutations, ...

Gene editing has made huge leaps in recent years, such as treating the congenital blood disorders sickle cell anemia and beta thalassemia, which can require lifelong blood transfusions. But scientists ...

A man with type 1 diabetes has become the first patient to produce his own insulin after receiving genetically engineered cell transplants, without needing drugs to prevent rejection. The case, ...