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 …

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 …

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 …

Background FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated
virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to …

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 …

Background The goal of gene therapy for patients with hemophilia A is to safely impart long-
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …

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 …

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 …

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …