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Repurposing the prostaglandin analogue treprostinil and the calcium-sensing receptor modulator cinacalcet to revive cord blood as an alternate source of hematopoietic stem and progenitor cells for transplantation.
Front Pharmacol
January 2025
Institute of Pharmacology and the Gaston H. Glock Research Laboratories for Exploratory Drug Development, Centre of Physiology and Pharmacology, Medical University of Vienna, Vienna, Austria.
Objective: The expanding field of hematopoietic cell transplantation (HCT) for non-malignant diseases, including those amenable to gene therapy or gene editing, faces challenges due to limited donor availability and the toxicity associated with cell collection methods. Umbilical cord blood (CB) represents a readily accessible source of hematopoietic stem and progenitor cells (HSPCs); however, the cell dose obtainable from a single cord blood unit is frequently insufficient. This limitation can be addressed by enhancing the potency of HSPCs, specifically their capacity to reconstitute hematopoiesis.
View Article and Find Full Text PDFLentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial.
Clin Transl Med
January 2025
Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Background: Fabry disease is an X-linked lysosomal storage disorder due to a deficiency of α-galactosidase A (α-gal A) activity. Our goal was to correct the enzyme deficiency in Fabry patients by transferring the cDNA for α-gal A into their CD34+ hematopoietic stem/progenitor cells (HSPCs). Overexpression of α-gal A leads to secretion of the hydrolase; which can be taken up and used by uncorrected bystander cells.
View Article and Find Full Text PDFImpact of Body Mass Index on Outcomes in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation Recipients: A Single-Center Retrospective Study.
Nutrients
October 2024
Department of Pediatrics, Institute for Maternal and Child Health-IRCCS Burlo Garofolo, 34137 Trieste, Italy.
Article Synopsis
- Pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) face risks related to their nutritional status, specifically body mass index (BMI), which affects transplantation success.
- A study analyzed BMI data from patients treated between 2003 and 2023 and found that underweight patients had significantly lower survival rates and higher complications compared to those with normal or higher BMI.
- The research emphasizes the need for regular nutritional assessment before and after transplant to improve outcomes, suggesting that BMI should be closely monitored to manage risks effectively.
Presence of fetal microchimerisms in the heart and effect on cardiac repair.
Front Cell Dev Biol
August 2024
Instituto de Investigación Sanitaria Gregorio Marañón (IiSGM), Madrid, Spain.
Multiple complex biological processes take place during pregnancy, including the migration of fetal cells to maternal circulation and their subsequent engraftment in maternal tissues, where they form microchimerisms. Fetal microchimerisms have been identified in several tissues; nevertheless, their functional role remains largely unknown. Different reports suggest these cells contribute to tissue repair and modulate the immune response, but they have also been associated with pre-eclampsia and tumor formation.
View Article and Find Full Text PDFGlucose intolerance as a consequence of hematopoietic stem cell dysfunction in offspring of obese mice.
Mol Metab
October 2024
Department of Pediatrics, Neonatal-Perinatal Medicine, Indiana University School of Medicine, Indianapolis, 46202, USA; Department of Anatomy, Cell Biology & Physiology, Indiana University School of Medicine, Indianapolis, 46202, USA; Center for Diabetes and Metabolic Disease, Indiana University School of Medicine, Indianapolis, 46202, USA; Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, 46202, USA. Electronic address:
Objective: Maternal obesity is increasingly common and negatively impacts offspring health. Children of mothers with obesity are at higher risk of developing diseases linked to hematopoietic system abnormalities and metabolism such as type 2 diabetes. Interestingly, disease risks are often dependent on the offspring's sex, suggesting sex-specific reprogramming effect of maternal obesity on offspring hematopoietic stem and progenitor cell (HSPC) function.
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