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.
Times cited: 75
Impaired intrinsic immunity to HSV-1 in human iPSC-derived TLR3-deficient CNS cells.
Times cited: 129