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Showing 1–8 of 8 results
Advanced filters: Author: Noah Reis Clear advanced filters
  • In an observational study evaluating functional precision medicine in children and adolescents with relapsed or refractory solid and hematologic malignancies, it was feasible to provide personalized treatment recommendations to treating physicians on the basis of genomic profiling and ex vivo drug sensitivity testing within 4 weeks.

    • Arlet M. Acanda De La Rocha
    • Noah E. Berlow
    • Diana J. Azzam
    ResearchOpen Access
    Nature Medicine
    Volume: 30, P: 990-1000
  • Antisense oligonucleotides effectively decrease the inclusion of exon  8A of CACNA1C in human cells both in vitro and in rodents transplanted with human brain organoids, and a single intrathecal administration rescued both calcium changes and in vivo dendrite morphology of patient neurons.

    • Xiaoyu Chen
    • Fikri Birey
    • Sergiu P. Pașca
    ResearchOpen Access
    Nature
    Volume: 628, P: 818-825
  • Assembloids are integrated with CRISPR screening to investigate the involvement of 425 neurodevelopmental disorder genes in human interneuron development, showing endoplasmic reticulum displacement before nuclear translocation and interference from LNPK deletion, resulting in abnormal migration.

    • Xiangling Meng
    • David Yao
    • Sergiu P. Pașca
    ResearchOpen Access
    Nature
    Volume: 622, P: 359-366
  • How mRNA-based coronavirus disease 2019 vaccines drive immune responses is not clear. Here the authors characterize immune responses to the BNT162b2 vaccine in mice, and show how it stimulates innate immunity, with antigen-specific CD8+ T cell responses dependent on the RNA sensor MDA5.

    • Chunfeng Li
    • Audrey Lee
    • Bali Pulendran
    Research
    Nature Immunology
    Volume: 23, P: 543-555
  • This protocol combines pooled CRISPR–Cas9 screening with neural organoid and assembloid models and illustrates how it can be applied to map hundreds of disease genes onto cellular pathways and specific aspects of human neural development.

    • Xiangling Meng
    • Noah Reis
    • Sergiu P. Pașca
    Protocols
    Nature Protocols
    P: 1-21