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 …

Gene therapy is a technique involving the modification of an individual's genes for treating a
particular disease. The key to effective gene therapy is an efficient carrier delivery system …

Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec
(AAV5-hFVIII-SQ) has demonstrated clinical benefits lasting 5 years to date in people with …

In the last decade, enormous progress has been made in the development of gene therapy
for hemophilia A and B. After the first encouraging results of intravenously administered …

Introduction Valoctocogene roxaparvovec is an investigational AAV5‐based factor VIII (FVIII)
gene therapy that has demonstrated sustained clinical benefit in people with severe …

Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …

Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Introduction Hemophilia comprises a group of X-linked hemorrhagic disorders that result
from a deficiency of coagulation factors. The disorder affects mainly males and leads to …

Gene editing ushers in a new era of disease treatment since many genetic diseases are
caused by base-pair mutations in genomic DNA. With the rapid development of genome …

Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …