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• W4385623793 abstract "Blood platelets have unique storage and delivery capabilities. Platelets play fundamental roles in hemostasis, inflammatory reactions, and immune responses. Beyond their functions, platelets have been used as a target for gene therapy. Platelet-targeted gene therapy aims to deliver a sustained expression of neo-protein in vivo by genetically modifying the target cells, resulting in a cure for the disease. Even though there has been substantial progress in the field of gene therapy, the potential development of immune responses to transgene products or vectors remains a significant concern. Of note, multiple preclinical studies using platelet-specific lentiviral gene delivery to hematopoietic stem cells in hemophilia have demonstrated promising results with therapeutic levels of neo-protein that rescue the hemorrhagic bleeding phenotype and induce antigen-specific immune tolerance. Further studies using ovalbumin as a surrogate protein for platelet gene therapy have shown robust antigen-specific immune tolerance induced via peripheral clonal deletions of antigen-specific CD4- and CD8-T effector cells and induction of antigen-specific regulatory T (Treg) cells. This review discusses platelet-targeted gene therapy, focusing on immune tolerance induction." @default.
• W4385623793 created "2023-08-08" @default.
• W4385623793 creator A5018476169 @default.
• W4385623793 creator A5026060508 @default.
• W4385623793 creator A5090807899 @default.
• W4385623793 date "2023-08-01" @default.
• W4385623793 modified "2023-10-14" @default.
• W4385623793 title "Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond" @default.
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