Platelet-Targeted FVIII Gene Therapy Restores Hemostasis and Induces Immune Tolerance for Hemophilia A. Front Immunol 2020;11:964
Date
07/01/2020Pubmed ID
32595633Pubmed Central ID
PMC7303294DOI
10.3389/fimmu.2020.00964Scopus ID
2-s2.0-85087185344 (requires institutional sign-in at Scopus site) 5 CitationsAbstract
Platelets are small anucleated blood components primarily described as playing a fundamental role in hemostasis and thrombosis. Over the last decades, increasing evidence has demonstrated the role of platelets in modulating inflammatory reactions and immune responses. Platelets harbor several specialized organelles: granules, endosomes, lysosomes, and mitochondria that can synthesize proteins with pre-stored mRNAs when needed. While the functions of platelets in the immune response are well-recognized, little is known about the potential role of platelets in immune tolerance. Recent studies demonstrate that platelet-specific FVIII gene therapy can restore hemostasis and induce immune tolerance in hemophilia A mice, even mice with preexisting anti-FVIII immunity. Here, we review the potential mechanisms by which platelet-targeted FVIII gene therapy restores hemostasis in the presence of anti-FVIII inhibitory antibodies and induces immune tolerance in hemophilia A.
Author List
Cai Y, Shi QAuthor
Qizhen Shi MD, PhD Professor in the Pediatrics department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AnimalsAntibodies
Blood Platelets
Factor VIII
Gene Targeting
Genetic Therapy
Hemophilia A
Hemostasis
Humans
Immune Tolerance
Treatment Outcome
