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.

The company is targeting liver and CNS disorders employing a array of tools. Their “Smart AAV” technology provides novel capsids for CNS delivery that utilize antibodies and peptides for ...

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 ...

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 ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft demand for gene therapies among haemophilia patients and physicians.

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 ...

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 ...

"The liver has this inherent regenerative capacity ... "Our approach used adeno-associated viruses (AAV) to deliver CRISPR/Cas9. However, we believe the Repair Drive platform has tremendous ...

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 ...