Search Results - "Sproul, Scott" :: K.UTB vyhledávací portál | TBU Library Retrieval Portal

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

by Sharma, Rajiv, Anguela, Xavier M., Doyon, Yannick, Wechsler, Thomas, DeKelver, Russell C., Sproul, Scott, Paschon, David E., Miller, Jeffrey C., Davidson, Robert J., Shivak, David, Zhou, Shangzhen, Rieders, Julianne, Gregory, Philip D., Holmes, Michael C., Rebar, Edward J., High, Katherine A.
Published in Blood (08.10.2015)

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AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction

AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction

by Yasuda, Makiko, Huston, Marshall W., Pagant, Silvere, Gan, Lin, St. Martin, Susan, Sproul, Scott, Richards, Daniel, Ballaron, Stephen, Hettini, Khaled, Ledeboer, Annemarie, Falese, Lillian, Cao, Liching, Lu, Yanmei, Holmes, Michael C., Meyer, Kathleen, Desnick, Robert J., Wechsler, Thomas
Published in Molecular therapy. Methods & clinical development (11.09.2020)

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ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

by Wechsler, Thomas, Meyer, Kathleen E., Spratt, S. Kaye, Greengard, Judith, Santiago, Yolanda, Sproul, Scott, Surosky, Richard, Paschon, David E., Dubois-Stringfellow, Nathalie, Ando, Dale Genji, Nichol, Geoff, Rebar, Edward J., Holmes, Michael C.
Published in Blood (03.12.2015)

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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

by Laoharawee, Kanut, DeKelver, Russell C., Podetz-Pedersen, Kelly M., Rohde, Michelle, Sproul, Scott, Nguyen, Hoang-Oanh, Nguyen, Tam, St. Martin, Susan J., Ou, Li, Tom, Susan, Radeke, Robert, Meyer, Kathleen E., Holmes, Michael C., Whitley, Chester B., Wechsler, Thomas, McIvor, R. Scott
Published in Molecular therapy (04.04.2018)

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ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

by Pagant, Silvere, Huston, Marshall W., Moreira, Luciana, Gan, Lin, St Martin, Susan, Sproul, Scott, Holmes, Michael C., Meyer, Kathleen, Wechsler, Thomas, Desnick, Robert J., Yasuda, Makiko
Published in Molecular therapy (03.11.2021)

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Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction

Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction

by Huston, Marshall W., Yasuda, Makiko, Pagant, Silvere, Martin, Susan St, Sproul, Scott, DeKelver, Russell, Holmes, Michael C., Desnick, Robert J., Wechsler, Thomas
Published in Molecular genetics and metabolism (01.02.2018)

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ZFN-mediated in vivo genome editing results in therapeutic levels of α-galactosidase A and effective substrate reduction in Fabry knockout mice

ZFN-mediated in vivo genome editing results in therapeutic levels of α-galactosidase A and effective substrate reduction in Fabry knockout mice

by Pagant, Silvere, Huston, Marshall W., Yasuda, Makiko, Martin, Susan St, Sproul, Scott, DeKelver, Russell, Holmes, Michael C., Wechsler, Thomas, Desnick, Robert J.
Published in Molecular genetics and metabolism (01.02.2018)

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Liver-based expression of the human alpha-galactosidase A gene (GLA) in a murine Fabry model results in continuous supra-physiological enzyme activity and effective substrate reduction

Liver-based expression of the human alpha-galactosidase A gene (GLA) in a murine Fabry model results in continuous supra-physiological enzyme activity and effective substrate reduction

by Huston, Marshall W., Yasuda, Makiko, Pagant, Silvere, St. Martin, Susan, Sproul, Scott, Santiago, Yolanda, DeKelver, Russel, Holmes, Michael C., Desnick, Robert, Wechsler, Thomas
Published in Molecular genetics and metabolism (01.01.2017)

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ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models

ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models

by 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.
Published in Molecular genetics and metabolism (01.02.2018)

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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

by 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.
Published in Molecular therapy (02.01.2019)

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484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)

484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)

by Laoharawee, Kanut, DeKelver, Russell, Podetz-Petersen, Kelly M., Tom, Susan, Radeke, Robert, Rohde, Michelle, Manning-Bog, Amy, Sproul, Scott, Ou, Li, Meyer, Kathleen, Holmes, Michael C., Whitley, Chester B., Wechsler, Thomas, McIvor, R. Scott
Published in Molecular therapy (01.05.2016)

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485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I

485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I

by 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.
Published in Molecular therapy (01.05.2016)

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ZFN-mediated genome editing of albumin “safe harbor” in vivo results in supraphysiological levels of human IDS, IDUA and GBA in mice

ZFN-mediated genome editing of albumin “safe harbor” in vivo results in supraphysiological levels of human IDS, IDUA and GBA in mice

by DeKelver, Russell, Rohde, Michelle, Tom, Susan, Santiago, Yolanda, Sproul, Scott, Gregory, Philip D., Holmes, Michael C., Wechsler, Thomas
Published in Molecular genetics and metabolism (01.02.2015)

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ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models

ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models

by 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
Published in Molecular genetics and metabolism (01.01.2017)

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Abstract 5609: Mechanisms of acquired resistance to selective PI3K inhibitors in breast cancer cell lines

Abstract 5609: Mechanisms of acquired resistance to selective PI3K inhibitors in breast cancer cell lines

by O'Brien, Carol Lynn, Spoerke, Jill, Lackner, Mark, Sproul, Scott, Haverty, Peter, Hampton, Garret
Published in Cancer research (Chicago, Ill.) (15.04.2012)

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479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease

479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease

by Wechsler, Thomas, DeKelver, Russel, Rohde, Michelle, Tom, Susan, Sproul, Scott, Ou, Leo, Laoharawee, Kanut, Podetz-Pedersen, Kelly M., Whitley, Chester B., McIvor, Scott R., Gregory, Philip D., Holmes, Michael C.
Published in Molecular therapy (01.05.2015)

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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

ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus

by 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.
Published in Molecular genetics and metabolism (01.02.2016)

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ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model

ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model

by DeKelver, Russell, Laoharawee, Kanut, Tom, Susan, Radeke, Robert, Rohde, Michelle, Manning-Bog, Amy, Sproul, Scott, Podetz-Pedersen, Kelly M., Holmes, Michael C., Whitley, Chester B., Scott McIvor, R., Wechsler, Thomas
Published in Molecular genetics and metabolism (01.02.2016)

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Abstract 3071: Accurate and sensitive detection of KRAS mutations in heterogeneous cancer specimens

Abstract 3071: Accurate and sensitive detection of KRAS mutations in heterogeneous cancer specimens

by Bao, Yun, Merrill, David, Corre, Delphine Le, Sproul, Scott, Ching, Bonnie, Casuga, Iris, Sapinoso, Lorenzo, Desai, Sejal, Petraroli, Rosella P., Deng, David, Brzoska, Pius, Blons, Helene, Laurent-Puig, Pierre, Chen, Caifu
Published in Cancer research (Chicago, Ill.) (15.04.2011)

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Or maybe before closing

Or maybe before closing

by Sproul, Scott
Published in Daily sentinel-review (Woodstock, 1968) (05.12.2018)

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