Liver International, Journal Year: 2024, Volume and Issue: unknown

Published: Sept. 9, 2024

Gene therapy is being successfully developed for the treatment of several genetic disorders. Various methods gene transfer have been to enable production deficient enzyme or protein. One most important adeno-associated virus vectors, which shown be viable use in vivo therapy. Several therapies already approved. They are also promising acquired diseases. Important examples include haemophilia A and B, X-linked myotubular myopathy, spinal muscular atrophy liver diseases such as Criggler-Najjar disease, alpha-1 antitrypsin deficiency Fabry disease. However, introduction a foreign compound into hepatocytes leads hepatic reactions with heterogeneous phenotypic expression wide spectrum severity, ranging from mild transaminase elevation acute failure. mechanisms appear involved injury, including an immune response, but direct toxicity depending on method transfer. As result, incidence, severity injury vary indication patient patient. Patients treated more prone than those B. Corticosteroids used correct prevent degradation transferred loss therapeutic activity. The aim this review describe risk according short- long-term management currently proposed clinical practice.

Language: Английский