ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome von Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michelle J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, Whitley, Chester B.

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Enzyme replacement therapy with α-L-iduronidase and lectin RTB fusion protein in treating murine Hurler syndrome von Ou, Li, Acosta, Walter, Koniar, Brenda L., Cooksley, Renee D., Cramer, Carole L., Radin, David N., Whitley, Chester B.

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ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models von Wechsler, Thomas, DeKelver, Russell, Laoharawee, Kanut, Ou, Li, Tom, Susan, Radecke, Robert, Sproul, Scott, Koniar, Brenda L., Podetz-Perdersen, Kelly, Cooksley, Renee D., Meyer, Kathleen, McIvor, Scott R., Chester, Whitley B., Holmes, Michael C.

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485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I von Ou, Li, DeKelver, Russell, Tom, Susan, Radeke, Robert, Rohde, Michelle, Manning-Bog, Amy, Sproul, Scott, Przybilla, Michael J., Komar, Brenda L., Podetz-Pedersen, Kelly, Laoharawee, Kanut, Cooksley, Renee D., Holmes, Michael C., Wechsler, Thomas, McIvor, R. Scott, Whitley, Chester B.

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ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models von DeKelver, Russell, Ou, Li, Laoharawee, Kanut, Tom, Susan, Radeke, Robert, Rohde, Michelle, Sproul, Scott, Przybilla, Michael, Koniar, Brenda L, Podetz-Pedersen, Kelly, Cooksley, Renee D, Holmes, Michael C, McIvor, R. Scott, Whitley, Chester B, Wechsler, Thomas

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ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus von Ou, Li, DeKelver, Russell, Tom, Susan, Radeke, Robert, Rhode, Michelle, Manning-Bog, Amy, Sproul, Scott, Przybilla, Michael J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Holmes, Michael C., Wechsler, Thomas, Scott McIvor, R., Whitley, Chester B.

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Buccal Swab Specimens for IDUA Genotype Analysis for Patients after Hematopoietic Stem Cell Transplantation von Cooksley, Renee D., Shapiro, Elsa, Orchard, Paul, Tolar, Jakub, Whitley, Chester B.

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28. Case studies in mutational analysis: Challenges remain von Cooksley, Renee, Sandberg, Sherri, Score, Paul, Diethelm-Okita, Brenda, Erickson, David, Whitley, Chet

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