Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.

Autor: Laoharawee K; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Podetz-Pedersen KM; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Nguyen TT; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Evenstar LB; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Kitto KF; 2 Department of Neuroscience and College of Pharmacy, University of Minnesota , Minneapolis., Nan Z; 3 Department of Neurosurgery, University of Minnesota , Minneapolis., Fairbanks CA; 2 Department of Neuroscience and College of Pharmacy, University of Minnesota , Minneapolis., Low WC; 3 Department of Neurosurgery, University of Minnesota , Minneapolis., Kozarsky KF; 4 REGENXBIO, Inc. , Rockville, Maryland., McIvor RS; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis.

Publikováno v: Human gene therapy [Hum Gene Ther] 2017 Aug; Vol. 28 (8), pp. 626-638. Date of Electronic Publication: 2017 May 05.