Category Ranking
98%
Total Visits
921
Avg Visit Duration
2 minutes
Citations
20
Article Abstract
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1038/s41409-024-02440-x | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches.
Front Pharmacol
August 2025
Department of Pharmacy, Faculty of Pharmacy and Health Sciences, Royal College of Medicine Perak, Universiti Kuala Lumpur, Ipoh, Malaysia.
Sickle cell disease (SCD) is an inherited blood disorder marked by the production of abnormal hemoglobin, leading to the distortion-or sickling-of red blood cells. The SCD arises from a single-point mutation that substitutes glutamic acid with valine at the sixth codon of the β-globin chain in hemoglobin. This substitution promotes deoxyhemoglobin aggregation, elevating red blood cell stiffness, and triggering vaso-occlusive and hemolytic repercussions.
View Article and Find Full Text PDFHematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia.
Mol Ther Methods Clin Dev
September 2025
Molecular Biology Interdepartmental Program, University of California, Los Angeles, Los Angeles, CA 90095, USA.
X-linked agammaglobulinemia (XLA) is a rare inborn error of immunity caused by loss-of-function mutations in the gene encoding Bruton's tyrosine kinase (BTK). XLA patients lack mature B cells and have negligible antibody levels, leaving them susceptible to recurrent bacterial and chronic viral infections. Autologous hematopoietic stem cell gene therapy with gene-corrected HSC may serve as a promising treatment of XLA; this therapy would provide a one-time cure and would replace lifelong immunoglobulin replacement therapy.
View Article and Find Full Text PDFAdoptive cellular therapies in multiple myeloma.
Best Pract Res Clin Haematol
September 2025
Department of Personalized Medicine and Rare Diseases, Medfuture Institute for Biomedical Research - Department of Hematology, Iuliu Hațieganu University of Medicine and Pharmacy, Cluj-Napoca, Romania; Department of Hematology, Ion Chiricuta Cancer Center, Cluj Napoca, Romania. Electronic address:
Plasma cell myeloma (multiple myeloma) is a blood cancer characterized by the clonal proliferation of plasma cells in the bone marrow. Treatment strategies evolve year by year, new drugs getting Food and Drug Administration (FDA)-approved each year. Chimeric antigen receptor (CAR) therapies are an advanced form of immunotherapy that engineer T cells to recognize and destroy cancer cells.
View Article and Find Full Text PDFAcute Respiratory Distress Syndrome Phenotypes After Stem Cell Transplantation: A Latent Class Analysis.
Crit Care Explor
September 2025
Division of Pulmonary and Critical Care Medicine, Mayo Clinic, Rochester, MN.
Objective: To identify distinct phenotypes of acute respiratory distress syndrome (ARDS) developing after hematopoietic cell transplantation (HCT), using routinely available clinical data at ICU admission.
Design: Multicenter retrospective cohort study using latent class analysis.
Setting: ICUs across three Mayo Clinic campuses (Minnesota, Florida, and Arizona).
Real-world effectiveness of autologous haematopoietic stem cell transplantation for multiple sclerosis in the UK.
J Neurol Neurosurg Psychiatry
September 2025
Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UK.
Background: Autologous haematopoietic stem cell transplantation (AHSCT) is increasingly used as a one-off disease-modifying therapy for aggressive forms of multiple sclerosis (MS). We report real-world effectiveness of AHSCT for MS in the UK.
Methods: This retrospective open-label study included patients with (pw)MS treated with AHSCT between 2002 and 2023 in 14 UK centres.