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Showing 1–2 of 2 results
Advanced filters: Author: Mary-Alice Abbott Clear advanced filters
  • A phase 1/2 trial of dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis, administered by bilateral intrathalamic, cisterna magna and intrathecal delivery found a dose-dependent biochemical correction of the disease.

    • Florian Eichler
    • Oguz I. Cataltepe
    • Terence R. Flotte
    ResearchOpen Access
    Nature Medicine
    Volume: 31, P: 2927-2935
  • Michael Talkowski and colleagues analyze balanced chromosomal abnormalities in 273 individuals by whole-genome sequencing. Their findings suggest that sequence-level resolution improves prediction of clinical outcomes for balanced rearrangements and provides insight into pathogenic mechanisms such as altered gene regulation due to changes in chromosome topology.

    • Claire Redin
    • Harrison Brand
    • Michael E Talkowski
    Research
    Nature Genetics
    Volume: 49, P: 36-45