Jessica Nance, MD, MS, discusses maximizing gene therapy efficiency by selecting vectors for gene size, tissue targeting, and low exposure, and focusing on precise promoters for expression. This is a ...

Explore strategies for improving recombinant adeno-associated virus production in transient HEK293 suspension cultures while maintaining vector quality and supporting scalable viral vector ...

Cell and gene therapies rely on engineered viruses to deliver them to their cellular destinations. While the progress of these types of medicines is evident in a growing number of FDA product ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental study, ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

Investigators headed by a team at Brigham and Women’s Hospital have developed an adeno-associated virus (AAV) vector that studies show can far more efficiently cross the blood-brain barrier (BBB), in ...

Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale ...

Children born with severe-to-profound hearing loss caused by mutations in the OTOF gene now have a treatment option that did not exist a week ago. On April 23, 2026, the FDA granted accelerated ...

We use the AdenoX Rapid Titer kit (Clontech now Takara Bio) for this service. This immunohistochemical assay involves making 3 serials dilutions of the virus and infecting 6 wells of a 24-well plate ...