Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa by Timothy K. MacLachlan, Mark N. Milton, Oliver Turner, Francis Tukov, Vivian W. Choi, Jan Penraat, Marie-Hélène Delmotte, Lydia Michaut, Bruce D. Jaffee, Chad E. Bigelow

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Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA by Nicolina Cristina Sorrentino, Vincenzo Cacace, Maria De Risi, Veronica Maffia, Sandra Strollo, Novella Tedesco, Edoardo Nusco, Noemi Romagnoli, Domenico Ventrella, Yan Huang, Nan Liu, Susan L. Kalled, Vivian W. Choi, Elvira De Leonibus, Alessandro Fraldi

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Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates by Nancy Chen, David E. Ehmann, Robert Crooker, Katayoun Derakhchan, Xiaodong Fang, Brian Felice, Elizabeth J. Galbreath, Charles Glaus, Hongbo Gu, Yan Huang, Christine Li, Xing Li, Nan Liu, Kathleen Palmieri, Damir Simic, Joseph Sypek, Susan Thompson, Christopher T. Winkelmann, Vivian W. Choi

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AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout mice by Vivian W Choi, Chad E Bigelow, Terri L McGee, Akshata N Gujar, Hui Li, Shawn M Hanks, Joanna Vrouvlianis, Michael Maker, Barrett Leehy, Yiqin Zhang, Jorge Aranda, George Bounoutas, John T Demirs, Junzheng Yang, Richard Ornberg, Yu Wang, Wendy Martin, Kelly R Stout, Gregory Argentieri, Paul Grosenstein, Danielle Diaz, Oliver Turner, Bruce D Jaffee, Seshidhar R Police, Thaddeus P Dryja

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