TY  - JOUR
A1  - Mücke, Marcus Maximilian
A1  - Fong, Sylvia
A1  - Foster, Graham R.
A1  - Lillicrap, David
A1  - Miesbach, Wolfgang
A1  - Zeuzem, Stefan
T1  - Adeno-associated viruses for gene therapy – clinical implications and liver related complications, a guide for hepatologists
T2  - Journal of hepatology
N2  - Gene therapy has garnered increasing interest over recent decades. Several therapies employing gene transfer mechanisms have been developed, and, of these, adeno-associated virus (AAV) vectors have demonstrated viability for use with in vivo gene therapy. Several AAV-based therapeutics have received regulatory approval in the last few years including those for retinal disease, spinal muscular atrophy or aromatic L-amino acid decarboxylase deficiency. Lately, with the introduction of novel liver-directed AAV vector-based therapeutics for the treatment of haemophilia A and B, gene therapy has attracted significant attention in the hepatology community, with the liver increasingly recognised as a target for gene therapy. However, the introduction of foreign DNA into hepatocytes is associated with a risk of hepatic reactions, with raised ALT (alanine aminotransferase) and AST (aspartate aminotransferase) being – so far – the most commonly reported side effects. The complete mechanisms underlying the ALT flairs remain to be determined and the long-term risks associated with these new treatments is not yet known. The liver community is increasingly being asked to support liver-directed gene therapy to mitigate potential liver associated harm. In this review, we focus on AAV vector-based gene therapy, shedding light on this promising technique and its remarkable success in haemophilia, with a special focus on hepatic complications and their management in daily clinical practice.
KW  - Gene therapy
KW  - Adeno associated virus
KW  - hepatitis
KW  - hemophilia
Y1  - 2024
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/79426
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-794261
SN  - 0168-8278
VL  - 80
IS  - 2
SP  - 352
EP  - 361
PB  - Elsevier
CY  - Amsterdam u.a.
ER  -