Cell-mediated immunity to naglu transgene following intracerebral gene therapy in children with mucopolysaccharidosis type iiib syndrome

HIGHLIGHTS

  • who: Marie-Lise Gougeon from the (UNIVERSITY) have published the paper: Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome, in the Journal: (JOURNAL)
  • how: Similar observations were made for the CD8+ T_cell compartment .

SUMMARY

    Preclinical studies of CNS-directed AAV administration in non-human primates have reported AAV-specific circulating neutralizing antibodies and transgene-specific IFN-g-producing T_cells. Immune responses in the CNS are common, despite its perception as a site of immune privilege, and microglia and astrocytes can engage in . . .

     

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