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Pyridoxine supplementation confers protection against SGPL1 R222Q variant sphingosine phosphate lyase insufficiency syndrome.
AEGIS reveals epitope- and clone-resolved convergence of CNS B and T cell autoreactivity in ROHHAD.
Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.
Temporal AI model predicts drivers of cell state trajectories across human aging.
Structural variants in human congenital heart disease disrupt distal genomic regulatory contacts of developmental genes.
Local Tumor Microenvironment Niches Correlate With Survival And Immunotherapy Response In Human Glioblastoma.
A Large Animal Model of Heritable Pulmonary Arterial Hypertension Using Gene-edited BMPR2 Sheep.
COUNTERING AGE-ASSOCIATED ALTERATIONS IN OLIGODENDROCYTE-DERIVED EXTRACELLULAR MATRIX REJUVENATES COGNITION.
Structure-guided design of a targeted autoantibody degrader for neurologic disease.
Pre-myelinating oligodendrocyte ADGRG1 is required for axon ensheathment and CNS myelin formation.