Prevention of neurocognitive deficiency in mucopolysaccharidosis Type II mice by central nervous system-directed, AAV9-mediated iduronate sulfatase gene transfer

Kanut Laoharawee, Kelly M. Podetz-Pedersen, Tam T. Nguyen, Laura B. Evenstar, Kelley F. Kitto, Zhenhong Nan, Carolyn A. Fairbanks, Walter C. Low, Karen F. Kozarsky, R. Scott Mcivor

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Medicine & Life Sciences