Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Background Moderate-to-severe hemophilia B is treated with lifelong, continuous
coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims …

Abstract Background Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-
associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII …

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

Gene therapy is a promising approach for hereditary deafness. We recently showed that
unilateral AAV1-hOTOF gene therapy with dual adeno-associated virus (AAV) serotype 1 …

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 …

This new edition of the World Federation of Hemophilia (WFH) guidelines for the
management of hemophilia comes at an exciting time in the evolution of the diagnosis and …

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 …

Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or
deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease …