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http://dx.doi.org/10.1016/j.ymthe.2025.07.054 | DOI Listing |
Am J Hematol
September 2025
Division of Hematology, Mayo Clinic Rochester, Rochester, Minnesota, USA.
Immunol Lett
September 2025
Pediatric Cell and Gene Therapy Research Centre, Gene, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Tehran, Iran. Electronic address:
Introduction: Recent advances in hematopoietic stem cell transplantation (HSCT) have improved clinical outcomes; however, various factors continue to influence HSCT success, especially vaccination in immunocompromised patients who receive vaccination at birth. While several studies have investigated the efficacy of vaccines in Chronic Granulomatous Disease (CGD) patients, the specific impact of vaccination on HSCT outcomes in these patients has not yet been studied. This study aimed to address an important gap in the current literature by investigating the effects of BCG vaccination on HSCT outcomes in patients with CGD.
View Article and Find Full Text PDFGaucher disease type 1 is a lysosomal storage disorder caused by mutations that reduce glucocerebrosidase activity, leading to glycolipid buildup, particularly in macrophages. To develop a curative approach, we established a high-efficiency genome editing platform for human and murine hematopoietic stem-progenitor cells using CRISPR/Cas9, recombinant adeno-associated virus serotype 6. To enhance homology-directed DNA repair while minimizing genotoxicity, we incorporated a new 53BP1 inhibitor, a ubiquitin variant that promotes DNA end resection and significantly increases editing efficiency.
View Article and Find Full Text PDFBone Marrow Transplant
August 2025
CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000, Lille, France.
Reduced intensity conditioning (RIC) is usually used for patients with myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (allo-HCT), particularly in the elderly or those with comorbidities. The impact of conditioning intensity on patients' outcome remains controversial with clinicians' subjective opinion/ experience remaining a major guide in choosing the intensity. Here, we compare RIC versus MAC in a large EBMT retrospective study in MDS patients aged ≥50 years undergoing allo-HCT between 2014 and 2018.
View Article and Find Full Text PDFBehav Res Ther
August 2025
Department of Psychology, UCLA, United States.
Presenting unpaired unconditional stimuli (US) during extinction has been shown to reduce the contextual renewal of conditional fear and to slow re-acquisition. The present study investigated whether this reduced return of fear is also observed if the intensity of the US presented during extinction is lower than that presented during acquisition. Three groups of participants (N = 121) were trained in a differential fear conditioning procedure that employed habituation, acquisition, extinction, renewal test, and re-acquisition phases.
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