Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in gene therapy using adeno-associated virus vectors (AAV), specifically ...

This is called a normothermic liver perfusion system. The CMRI team has now demonstrated the potential of using this system to test AAV-based therapeutics prior to initiation of clinical studies.

Ferriero et al. show that liver fibrosis reduces the efficiency of AAV-based gene therapy by limiting liver uptake and altering vector distribution to other organs. The impact of fibrosis varies ...

A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.

The "AAV Vectors in Gene Therapy - Pipeline Insight, 2025" report has been added to ResearchAndMarkets.com's offering.The Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline signifies a ...

There is a growing list of approved AAV-based gene therapies and hundreds more in the clinical ... Of particular concern is the virus’s liking for the liver. “If you look at the number of vector ...

The two deaths occurred in children in Russia and Kazakhstan, with the liver failure starting five to six weeks after administration of the adeno-associated virus-based treatment. Both children ...

Most recently, he has been working on optimizing AAV vectors for haemophilia B and for liver-directed gene therapies, and has led pre-clinical studies on rare diseases including Niemann Pick ...

In both studies, adeno-associated virus 2 (AAV2) was detected in cases that required transplants as well as the vast majority of hospitalized cases, but was detected in very few of the matched healthy ...

Some research has suggested systemic administration of AAV vectors can cause damage to liver cells and inflammation. Astellas has since reported an interim readout from a phase 1/2 trial of its ...