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 …

Abstract Background Patients with the Crigler–Najjar syndrome lack the enzyme uridine
diphosphoglucuronate glucuronosyltransferase 1A1 (UGT1A1), the absence of which leads …

An outbreak of acute hepatitis of unknown aetiology in children was reported in Scotland in
April 2022 and has now been identified in 35 countries. Several recent studies have …

Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

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 …

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 …

Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …