Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …

The development of liver-based adeno-associated virus (AAV) gene therapies is facing
concerns about limited efficiency and durability of transgene expression. We evaluated …

Background Adeno-associated virus (AAV)–mediated gene therapy is under investigation as
a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years …

In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Background Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in
joints, soft tissue, and the central nervous system. Although successful gene transfer has …

In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic
disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served …

The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

Neutralizing antibodies to adeno-associated virus (AAV) vectors are highly prevalent in
humans,, and block liver transduction,–and vector readministration; thus, they represent a …

Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in
patients with hemophilia B by delivering a functional human F9 gene into liver cells. This …