Results 151 to 160 of about 217,775 (266)
ATP2B1 expression identifies human hematopoietic stem cells with superior repopulation and self-renewal. [PDF]
Varesi A +18 more
europepmc +1 more source
Human Monocytic Models Reveal Genotype‐Dependent Inflammatory Programs in VEXAS Syndrome
Objective VEXAS syndrome is a severe X‐linked autoinflammatory disorder caused by somatic mutations in ubiquitin‐like modifier activating enzyme 1 (UBA1), with clinical outcomes that vary by UBA1 genotype. We aimed to elucidate genotype‐specific inflammatory programs and identify potential therapeutic targets.
Kana Higashitani +10 more
wiley +1 more source
Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies. [PDF]
O'Hanlon Cohrt K, O'Dea S.
europepmc +1 more source
Precision medicine in paediatrics: Progress and priorities
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
CHEK2 loss endows chemotherapy resistance to hematopoietic stem cells. [PDF]
Zhou J +6 more
europepmc +1 more source
Affinity Peptides With pH Sensitivity for the Enrichment of CD38+ Cells
ABSTRACT The selective enrichment of cell populations based on surface markers is critical for the advancement of gene and cell therapies. Current antibody‐based cell isolation methods, such as fluorescence‐ and magnetic‐activated cell sorting (FACS and MACS), offer high specificity but are limited by scalability, cost, and potential adverse effects on
Gabrielle Rusch +7 more
wiley +1 more source
Single-cell vector copy number analysis of phenotypically defined long-term hematopoietic stem cells for gene therapy safety assessment. [PDF]
Sato Y +3 more
europepmc +1 more source
ABSTRACT Generation of CAR‐T cell therapies relies predominantly on lentiviral vectors (LVs). As in vivo CAR‐T trials progress, demand for larger quantities of higher‐potency LV is expected. However, primary capture by anion‐exchange chromatography (AIEX) remains a major manufacturing bottleneck from use of adsorbents not tailored to LVs, leading to ...
George Pamenter +10 more
wiley +1 more source

