Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes ...

Over the past few decades, there has been remarkable progress in genetic manipulation technologies, bringing us closer to the point where genes can be modified in vivo. Such tools would open up the ...

Pancreatitis is a complex inflammatory disease driven by both genetic and environmental factors. It poses substantial ...

Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

Researchers are making strides in improving gene therapies for genetic diseases, particularly chronic kidney disease, using adeno-associated virus, or AAV, vectors. While AAV-based treatments have ...

Researchers develop a nanosensing platform that can assess the quality of individual viral vector particles Viral vectors hold much potential for gene editing and gene therapy, but there is a pressing ...

Circio Holding has presented preclinical data at ASGCT demonstrating that its circVec circular RNA platform achieved up to 40 ...

A planned first-in-human trial aims to treat GM1 gangliosidosis in utero, possibly laying the foundation for prenatal gene ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...