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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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Beyond transaminitis: liver biopsy redefines human gene therapy hepatotoxicity

Wolfgang Miesbach1, Vincenzo Cardinale2

  • 1Department of Coagulation Disorders/Haemophilia Centre, Medical Clinic 2, University Hospital Frankfurt, Frankfurt.

Haematologica
|August 18, 2026
PubMed

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Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...