Transfus Apher Sci
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Sickle cell disease (SCD) is the most common monogenic blood disorder marked by severe pain, end-organ damage, and early mortality. Treatment options for SCD remain very limited. There are only four FDA approved drugs to reduce acute complications. ⋯ Ex vivo engineering of autologous hematopoietic stem and progenitor cells followed by transplantation of genetically modified cells potentially provides a permanent cure applicable to all patients regardless of the availability of suitable donors and graft-vs-host disease. In this review, we focus on the use of CRISPR/Cas9 gene-editing for curing SCD, including the curative correction of SCD mutation in β-globin (HBB) and the induction of fetal hemoglobin to reverse sickling. We summarize the major achievements and challenges, aiming to provide a clearer perspective on the potential of gene-editing based approaches in curing SCD.
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Passive antibody therapy has been used to immunize vulnerable people against infectious agents. In this study, we aim to investigate the efficacy of convalescent plasma (CP) in the treatment of severe and critically ill patients diagnosed with COVID-19. ⋯ CP therapy seems to be effective for a better course of COVID-19 in severe and critically ill patients.
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Coronavirus disease 2019 (COVID-19) is a disease spreading rapidly in Sudan, the rest of the African continent and the world with no known definitive treatment or vaccines. However, among many treatment interventions being tested globally, beneficial effects and clinical improvements have been reported when convalescent plasma is used for treating COVID-19 patients. We prepared a guiding protocol for treating early to moderate COVID-19 patients with plasma transfusion from convalescent COVID-19 patients. ⋯ The protocol covers instructions on patient and donor selection criteria, plasma harvesting, plasma product specifications, dosage and precautions for convalescent plasma collection and transfusion process. Altogether, we prepared a treatment protocol that is tailored to the context of Sudan to be adopted by Sudan's health authority. Moreover, it will also provide reference for researchers to design open label clinical trials for convalescent plasma transfusion.
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The Hematology Department and its Hematopoietic Cell Transplantation (HCT) program implemented several measures during COVID-19 outbreak in order to keep clinical activities with the maximum security for both donors and recipients. Nevertheless, there was a lack of evidence whether blood products and specifically bone marrow can cause transfusion-transmitted infection. Initially, there were many uncertainties and did not exist formal recommendations. ⋯ In our knowledge this is the first case report of an adult hematopoietic cell donor with COVID-19 in the incubation period where the transplant is successfully completed with no transmission of SARS-CoV-2. The low concentration of viral RNA in plasma of patients with COVID-19 could support the safety of blood products, including peripheral blood hematopoietic cells. In conclusion, blood products including hematopoietic stem cells are safe in the context of COVID-19 pandemic.