A Commemorative Issue in Honor of Professor Valder R. Arruda: Hemophilia and Rare Bleeding Disorders
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Biology".
Deadline for manuscript submissions: closed (31 August 2022) | Viewed by 3468
Special Issue Editor
Interests: hemophilia; rare bleeding disorders
Special Issue Information
Dear Colleagues,
Rare bleeding disorders (RBD) and hemophilia are inherited coagulopathies with variable bleeding phenotypes, whose hemostatic control was, until recently, based upon replacement therapy. In patients with bleeding symptoms, laboratory assessment and, especially, molecular workup enable an accurate diagnosis, as well as prenatal and family counseling. While future gene therapy may be promising for such patients, there is currently an unmet need to provide better functional tools (therapeutic options) for bleeding control and prevention, risk assessment directed at complications, e.g., unwanted immune responses to the therapeutic protein (inhibitor formation), and to address the use of non-replacement therapies as well as novel global assays that may be applied for monitoring. In advance phase studies on gene therapy for hemophilia, the control of critical safety and efficacy challenges will pave the way for approval as well as to set up the basis for future gene-based strategies for RBD. In this Special Issue, Molecular and Functional Research in Hemophilia and Rare Bleeding Disorders, we aim to discuss some of these important aspects associated with diagnosis and potential care.
Prof. Dr. Gili Kenet
Guest Editor
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Keywords
- hemophilia
- inhibitors
- rare bleeding disorders
- hemostasis
- gene therapy
- AAV
- lentiviral
- coagulation factors
- non-replacement therapy
- thrombotic complications
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