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Age-dependent changes in circulating Tfh cells influence development of functional malaria antibodies in children.
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency.
Common genetic variation associated with Mendelian disease severity revealed through cryptic phenotype analysis.
Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
The transcriptional landscape of Shh medulloblastoma.
Joint profiling of DNA and proteins in single cells to dissect genotype-phenotype associations in leukemia.
Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome.
Exposure to pesticides in utero impacts the fetal immune system and response to vaccination in infancy.
SARS-CoV-2 seroprevalence and neutralizing activity in donor and patient blood.
Defining the ATPome reveals cross-optimization of metabolic pathways.