Tag Archives: gene

Blind Man’s Sight Partially Restored with ‘Optogenetics’ Gene Therapy

Advertisement Continue reading the main story Supported by Continue reading the main story Matter Scientists Partially Restored a Blind Man’s Sight With New Gene Therapy Using a technique called optogenetics, researchers added light-sensitive proteins to the man’s retina, giving him a blurry view of objects. In an experiment, a 58-year-old volunteer with a genetic form… Read More »

New Zolgensma ‘inflection point’ is here as Novartis snags EU nod for SMA gene therapy

As the number of U.S. spinal muscular atrophy (SMA) patients Zolgensma treats each quarter stabilizes, Novartis is counting on a set of “inflection points” for future growth. Now, it has one. After a manufacturing-related delay, Novartis has won conditional approval in the EU for the one-time gene therapy to treat patients with a clinical diagnosis… Read More »

Modifier Gene May Explain Why Some with Cystic Fibrosis are Less Prone to Infection

Cystic fibrosis is caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Due to this mutation, the CFTR protein doesn’t embed in cell membranes to form a channel for chlorine ions the way it should. As a result, mucus-producing cells secrete a thicker-than-normal mucus that can create blockages in the lungs and… Read More »

FDA says some data testing Novartis’ $2 million gene therapy was manipulated

NEW YORK (Reuters) – The U.S. Food and Drug Administration said on Tuesday that some data from early testing of Novartis’ more than $ 2 million gene therapy Zolgensma was manipulated, but the agency believes the treatment should remain on the market. FILE PHOTO: The headquarters of the U.S. Food and Drug Administration (FDA) is… Read More »