Mitochondrial diseases affect approximately 1 in 5,000 people worldwide, causing debilitating symptoms ranging from muscle weakness to stroke-like episodes.

Researchers have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI) cancers. The results show ...

The only remaining autonomous 'jumping gene' can only attach to, and stitch a copy of itself into, DNA when it builds up into large clusters and only as cells divide.

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing ...

As scientists explore treating genetic disorders before birth, the promise of foetal gene therapy is growing—but there are ...

ScaleReady, in collaboration with Wilson Wolf Manufacturing, Bio-Techne Corporation and CellReady, today announced that March Biosciences has been awarded a $200,000 G-Rex® Grant. March Biosciences' G ...

Hepatocellular carcinoma (HCC), the most common form of liver cancer, continues to pose a significant global health challenge ...

Multidisciplinary coordination is crucial for effective gene therapy administration in Duchenne ... They break the process down into 4 categories: institutional readiness, patient selection ...

Stocktwits - Shares of gene therapy developer uniQure N.V. (QURE) shot up 41% on Thursday afternoon after it announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy ...

High dose and low dose AMT-130 demonstrated an 80% and 30% slowing of disease progression vs external control, respectively. The Food and Drug Administration has granted Breakthrough Therapy ...

Also Read: uniQure/ CSL Behring’s Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years According to a 2021 study in Neuroepidemiology, approximately 70,000 people have been ...

Japanese pharmaceutical company Takeda and domestic biotech company Belief BioMed jointly announced on Thursday that the gene therapy drug Dalnacogene Ponparvovec Injection has received approval ...