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http://dx.doi.org/10.1007/s10875-020-00855-x | DOI Listing |
PLoS Comput Biol
September 2025
Department of Genome Sciences, University of Washington, Seattle, Washington, United States of America.
Fanconi Anemia (FA) is a heritable syndrome characterized by DNA damage repair deficits, frequent malformations and a significantly elevated risk of bone marrow failure, leukemia, and mucosal head and neck squamous cell carcinomas (HNSCC). Hematopoietic stem cell gene therapy can prevent marrow failure and lower leukemia risk, but mucosal gene therapy to lower HNSCC risk remains untested. Major knowledge gaps include an incomplete understanding of how rapidly gene-corrected cellular lineages could spread through the oral epithelium, and which delivery parameters are critical for ensuring efficient gene correction.
View Article and Find Full Text PDFJ Pediatr Hematol Oncol
September 2025
Children's Hospital of Michigan, Division of Hematology/Oncology.
Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet disorder characterized by abnormalities in platelet aggregation, resulting from quantitative or qualitative defects in integrins αIIb and β3. Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only potentially curative therapeutic approach for severe GT. In this report, we present 2 children with GT that underwent successful allo-HSCT, along with 2008 to 2022 data from the Center for International Blood and Marrow Transplant Research and a summary of the existing literature providing further evidence that allo-HSCT can be a curative approach that prevents severe and life-threatening bleeding in GT.
View Article and Find Full Text PDFPLoS Pathog
September 2025
Department of Virology, Immunology, and Microbiology, Boston University Chobanian & Avedisian School of Medicine, Boston, Massachusetts, United States of America.
While human autopsy samples have provided insights into pulmonary immune mechanisms associated with severe viral respiratory diseases, the mechanisms that contribute to a clinically favorable resolution of viral respiratory infections remain unclear due to the lack of proper experimental systems. Using mice co-engrafted with a genetically matched human immune system and fetal lung xenograft (fLX), we mapped the immunological events defining successful resolution of SARS-CoV-2 infection in human lung tissues. Viral infection is rapidly cleared from fLX following a peak of viral replication, histopathological manifestations of lung disease and loss of AT2 program, as reported in human COVID-19 patients.
View Article and Find Full Text PDFInt J Hematol
September 2025
Department of Hematology and Oncology, Asahikawa Red Cross Hospital, 1-1 Akebono-Cho, Asahikawa, Hokkaido, Japan.
Introduction: Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) requires reliable vascular access for medication, transfusion, and blood sampling, which often involves painful venipuncture. This prospective study evaluated a novel dual peripherally inserted central venous catheter (PICC) technique to reduce venipuncture frequency in allo-HSCT recipients.
Methods: The study enrolled 29 allo-HSCT recipients.