Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cdnas in children with mucopolysaccharidosis type IIIA disease: Results of a phase I/II trial.
Human Gene Therapy.
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Impaired intrinsic immunity to HSV-1 in human iPSC-derived TLR3-deficient CNS cells.
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