Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia

Helen Beard, Leanne Winner, Andrew Shoubridge, Emma Parkinson-Lawrence, Adeline A. Lau, Siti N. Mubarokah, Tabitha-Rose Lance, Barbara King, William Scott, Marten F. Snel, Paul J. Trim, Kim M. Hemsley

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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science