Restore Vision, Inc. ("Restore Vision"), a clinical-stage biotechnology company advancing gene therapies for retinal ...

To design safe clinical trials, it is crucial to better understand and predict gene editing outcomes in preclinical studies. Bence György and collaborators have shown that adeno-associated viruses ...

Several quality attributes (QAs) must be monitored to ensure an effective and safe final product when developing adeno-associated virus (AAV) vectors as drug products. Thermal stability, aggregate ...

Gene delivery vehicles have helped realize the concept of treating human diseases by introducing normal alleles of genes into appropriate target cells. These gene delivery vehicles include recombinant ...

Adeno-associated virus (AAV) has emerged as the most flexible and useful vector for gene therapy. With hundreds of gene therapy trials ongoing, safe and efficient production of AAV is absolutely ...

In recent years, adeno-associated virus (AAV) has been recognized as the leading vehicle (vector) for in vivo delivery of therapeutic genes because it is non-pathogenic and efficiently targets many ...

Gene therapy adeno-associated viruses (AAVs)—viruses that can be engineered to deliver DNA to target cells—are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to ...