The complex, confusing and poorly understood immune responses to AAV-mediated gene transfer in haemophilia-Is more or less immunosuppression required?
J Viral Hepat
; 31 Suppl 1: 21-25, 2024 04.
Article
em En
| MEDLINE
| ID: mdl-38606938
ABSTRACT
Attempts to achieve a functional cure or amelioration of the severe X linked bleeding disorders haemophilia A (factor VIII deficiency) and haemophilia B (factor IX deficiency) using AAV-based vectors have been frustrated by immune responses that limit efficacy and durability. The immune responses include adaptive and innate pathways as well as cytokine mediated inflammation, especially of the target organ cells-hepatocytes. Immune suppression has only been partly effective in clinical trials at ameliorating the immune response and the lack of good animal models has delayed progress in identifying mechanisms and developing more effective approaches to controlling these effects of AAV gene transfer. Here we discuss the arguments for and against more potent immunosuppression to improve factor expression after AAV-mediated gene therapy.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Hemofilia B
/
Hemofilia A
Limite:
Animals
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article