A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS gene therapy in Canavan mice

Seemin Seher Ahmed, Huapeng Li, Chunyan Cao, Elif M. Sikoglu, Andrew R. Denninger, Qin Su, Samuel Eaton, Ana A. Liso Navarro, Jun Xie, Sylvia Szucs, Hongwei Zhang, Constance Moore, Daniel A. Kirschner, Thomas N. Seyfried, Terence R. Flotte, Reuben Matalon, Guangping Gao

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Biochemistry, Genetics and Molecular Biology

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Neuroscience