A single IV injection of a microRNA-based biologic developed by Guangping Gao, Ph.D., Robert H. Brown Jr., DPhil, MD, Jun Xie ...

Adeno-associated virus (AAV)-based viral vectors used in human gene therapy can trigger innate immune pathways, leading to the initiation of adaptive immune responses. A new review article published ...

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, ...

Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression of the high-activity factor IX ...

Viral vector service expansion empowers scientists by offering high-quality research reagents through on-demand AAV production from Addgene’s vast plasmid repository WATERTOWN, Mass.--(BUSINESS WIRE)- ...

A preclinical study found that a single gene therapy injection slowed ALS progression, protected motor neurons, and extended ...

Viral vector release testing requires a battery of analytical methods that, for the most part, did not exist a decade ago, and the regulatory bar rises with every new gene therapy approval. For ...

Improving the therapeutic quality and consistency of cell and gene therapies requires the adoption of enhanced Critical Quality Attributes (CQAs) throughout the development and manufacturing processes ...

In the mid-1990s, Hiroyuki Nakai, now an adeno-associated virus (AAV) researcher at the Oregon Health and Science University, moved to the United States. Nakai quickly became fascinated with the ...

Epilepsy affects over 50 million people worldwide, making it one of the most common neurological disorders. Although ...