Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and accurate ...

Up to 60% of congenital and early-onset hearing loss is caused by genetic mutations in an inherited gene, and gene therapy ...

Adeno-associated virus (AAV) gene therapies hold great promise, but ensuring product quality is critical. One major challenge? The presence of empty capsids, which can reduce therapeutic effectiveness ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

Researchers have developed a DNA-based therapy that targets the PCSK9 gene to lower cholesterol naturally. Using polypurine ...

Over the last four years, Shriners Children’s St. Louis researchers have been working to develop a new way to prevent the effects of childhood obesity. Now, using gene therapy, Shriners Children’s St.

The cell and gene therapy drug delivery market plays a pivotal role in advancing precision medicine by ensuring the effective introduction of living cells and genetically modified genes into patients.

The agency will consider a submission that includes pooled data from ongoing studies, a decision analysts viewed as a notable ...

Up to 60% of congenital and early-onset hearing loss is caused by genetic mutations in an inherited gene, and gene therapy ...