Tag Archives: gene

PNA-based technique an essential part of the gene editing toolkit

In an article published in the April 8 issue of Nature, the National Institutes of Health’s Somatic Cell Gene Editing Consortium provided a detailed update on the progress of their nationwide effort to develop safer and more effective methods to edit the genomes of disease-relevant somatic cells and reduce the burden of disease caused by… Read More »

FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy

FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy Print this page May 24, 2019 — The U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe… Read More »

Second death in Novartis gene therapy trials under investigation

(Reuters) – Novartis AG, which this week announced positive interim trial results for its experimental gene therapy for spinal muscular atrophy, on Friday said investigation is underway into whether a second trial death could be related to the treatment. The logo of Swiss drugmaker Novartis AG is seen at its headquarters in Basel, Switzerland January… Read More »

More than 100 scientists in China say baby gene editing is 'crazy'

SHANGHAI (Reuters) – More than 100 scientists, most of them in China, have condemned as “crazy” and unethical altering human genes after a geneticist claimed he had changed the genes of twin girls to create the first gene-edited babies. Scientist He Jiankui shows “The Human Genome”, a book he edited, at his company Direct Genomics… Read More »