Search Results - "Handyside, Britta"

Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers by Sihn, Choong-Ryoul, Handyside, Britta, Liu, Su, Zhang, Lening, Murphy, Ryan, Yates, Bridget, Xie, Lin, Torres, Richard, Russell, Chris B., O'Neill, Charles A., Pungor, Erno, Bunting, Stuart, Fong, Sylvia

“…Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype 5 (AAV5)-based gene therapy vector containing a B-domain-deleted human…”
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Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter by Fong, Sylvia, Handyside, Britta, Sihn, Choong-Ryoul, Liu, Su, Zhang, Lening, Xie, Lin, Murphy, Ryan, Galicia, Nicole, Yates, Bridget, Minto, Wesley C., Vitelli, Catherine, Harmon, Danielle, Ru, Yuanbin, Yu, Guoying Karen, Escher, Claudia, Vowinckel, Jakob, Woloszynek, Jill, Akeefe, Hassib, Mahimkar, Rajeev, Bullens, Sherry, Bunting, Stuart

“…Adeno-associated virus 5 (AAV5)-human factor VIII-SQ (hFVIII-SQ; valoctocogene roxaparvovec) is an AAV-mediated product under evaluation for treatment of…”
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Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action by Liu, Su, Razon, Lisa, Ritchie, Olivia, Sihn, Choong-Ryoul, Handyside, Britta, Berguig, Geoffrey, Woloszynek, Jill, Zhang, Lening, Batty, Paul, Lillicrap, David, Agrawal, Vishal, Cortesio, Christa, Gebretsadik, Kahsay, Akeefe, Hassibullah, Colosi, Peter, Kim, Benjamin, Bunting, Stuart, Fong, Sylvia

“…Recombinant adeno-associated virus (AAV) is an effective platform for therapeutic gene transfer; however, tissue-tropism differences between species are a…”
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Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood by Zhang, Lening, Yates, Bridget, Murphy, Ryan, Liu, Su, Xie, Lin, Handyside, Britta, Sihn, Choong-Ryoul, Bouwman, Taren, Galicia, Nicole, Tan, Danielle, Fonck, Carlos, Arens, Jeremy, Clark, Annie, Zhang, Weiming, Chandra, Sundeep, Srimani, Jaydeep, Holcomb, Jennifer, Van Tuyl, Andrea, Henshaw, Joshua, Vettermann, Christian, Siso, Silvia, Su, Cheng, Bullens, Sherry, Bunting, Stuart, O’Neill, Charles, Fong, Sylvia

“…Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) gene transfer provided reduced bleeding for adult clinical trial participants with severe hemophilia A. However,…”
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Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec by Zhang, Lening, Handyside, Britta, Murphy, Ryan, Sihn, Choong-Ryoul, Xie, Lin, Vitelli, Catherine, Harmon, Danielle, Sisó, Sílvia, Liu, Su, Bullens, Sherry, Bunting, Stuart, Fong, Sylvia

“…AAV5-hFVIII-SQ (valoctocogene roxaparvovec) is an adeno-associated virus (AAV)-mediated gene therapy vector containing a B-domain-deleted human factor VIII…”
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Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells by Handyside, Britta, Ismail, Ashrafali Mohamed, Zhang, Lening, Yates, Bridget, Xie, Lin, Sihn, Choong-Ryoul, Murphy, Ryan, Bouwman, Taren, Kim, Chan Kyu, De Angelis, Rolando, Karim, Omair A., McIntosh, Nicole L., Doss, Michael Xavier, Shroff, Shilpa, Pungor, Erno, Bhat, Vikas S., Bullens, Sherry, Bunting, Stuart, Fong, Sylvia

“…Recombinant adeno-associated virus (rAAV) vectors are often produced in HEK293 or Spodoptera frugiperda (Sf)-based cell lines. We compared expression profiles…”
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Interim 52-week analysis of immunogenicity to the vector capsid and transgene-expressed human FVIII in GENEr8-1, a phase 3 clinical study of valoctocogene roxaparvovec, an AAV5-mediated gene therapy for hemophilia A by Long, Brian, Fong, Sylvia, Handyside, Britta, Robinson, Tara, Day, Jonathan, Yu, Hua, Lau, Kelly, Patton, Kathryn, de Hart, Greg, Henshaw, Josh, Agarwal, Suresh, Vettermann, Christian, Gupta, Soumi

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Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice by Bunting, Stuart, Zhang, Lening, Xie, Lin, Bullens, Sherry, Mahimkar, Rajeev, Fong, Sylvia, Sandza, Krystal, Harmon, Danielle, Yates, Bridget, Handyside, Britta, Sihn, Choong-Ryoul, Galicia, Nicole, Tsuruda, Laurie, O’Neill, Charles A., Bagri, Anil, Colosi, Peter, Long, Shinong, Vehar, Gordon, Carter, Barrie

“…Hemophilia A is an X-linked bleeding disorder caused by mutations in the gene encoding the factor VIII (FVIII) coagulation protein. Bleeding episodes in…”
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A small molecule chaperone for alpha-1 antitrypsin deficiency-associated liver disease reduces liver polymer burden in the PiZ mouse model by Handyside, Britta, Zhang, Lening, Ngo, Katina, Murphy, Ryan, Chen, Joseph, Galicia, Nicole, Gorostiza, Olivia, Pacheco, Glenn, Xie, Lin, Mackenzie, Donald, Jones, Heidi, Heglar, Brian, Wang, Bing, Bhagwat, Shripad, Lomas, David, Irving, James, Ronzoni, Riccardo, Bullens, Sherry, Fong, Sylvia, Bunting, Stuart

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Abstract 12927: Impact of AAV- MYBPC3 Gene Transfer on Heart Structure and Function in Human and Mouse Models of Hypertrophic Cardiomyopathy by Vitelli, Catherine, Zhang, Lening, Murphy, Ryan, Blus, Bartlomiej, Christianson, Terri, Giaramita, Alexander, Holtzinger, John, Handyside, Britta, May, Hannah, Pham, Ann, Nguyen, Tu, Zhou, Huiyu, Ntai, Ioanna, Mearini, Giulia, Ricotti, Valeria, Voit, Thomas, Eschenhagen, Thomas, Carrier, Lucie, Agarwal, Pooja, Bunting, Stuart, Sumandea, Marius

“…Abstract only Background: Hypertrophic cardiomyopathy (HCM) is a life-threatening inherited heart disease characterized by left ventricular hypertrophy and…”
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Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice by Chen, Joseph C., Luu, Amanda R., Wise, Nathan, Angelis, Rolando De, Agrawal, Vishal, Mangini, Linley, Vincelette, Jon, Handyside, Britta, Sterling, Harry, Lo, Melanie J., Wong, Hio, Galicia, Nicole, Pacheco, Glenn, Van Vleet, Jeremy, Giaramita, Alexander, Fong, Sylvia, Roy, Sushmita M., Hague, Chuck, Lawrence, Roger, Bullens, Sherry, Christianson, Terri M., d'Azzo, Alessandra, Crawford, Brett E., Bunting, Stuart, LeBowitz, Jonathan H., Yogalingam, Gouri

“…Autosomal recessive mutations in the galactosidase β1 (GLB1) gene cause lysosomal β-gal deficiency, resulting in accumulation of galactose-containing…”
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Intermittent enzyme replacement therapy with recombinant human β-galactosidase prevents neuraminidase 1 deficiency by Luu, Amanda R., Wong, Cara, Agrawal, Vishal, Wise, Nathan, Handyside, Britta, Lo, Melanie J., Pacheco, Glenn, Felix, Jessica B., Giaramita, Alexander, d'Azzo, Alessandra, Vincelette, Jon, Bullens, Sherry, Bunting, Stuart, Christianson, Terri M., Hague, Charles M., LeBowitz, Jonathan H., Yogalingam, Gouri

“…Mutations in the galactosidase β 1 (GLB1) gene cause lysosomal β-galactosidase (β-Gal) deficiency and clinical onset of the neurodegenerative lysosomal storage…”
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Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction by Magat, Jenna, Jones, Samantha, Baridon, Brian, Agrawal, Vishal, Wong, Hio, Giaramita, Alexander, Mangini, Linley, Handyside, Britta, Vitelli, Catherine, Parker, Monica, Yeung, Natasha, Zhou, Yu, Pungor, Erno, Slabodkin, Ilya, Gorostiza, Olivia, Aguilera, Allora, Lo, Melanie J., Alcozie, Saida, Christianson, Terri M., Tiger, Pascale M.N., Vincelette, Jon, Fong, Sylvia, Gil, Geuncheol, Hague, Chuck, Lawrence, Roger, Wendt, Daniel J., Lebowitz, Jonathan H., Bunting, Stuart, Bullens, Sherry, Crawford, Brett E., Roy, Sushmita M., Woloszynek, Josh C.

“…Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder caused by N-sulfoglucosamine sulfohydrolase (SGSH) deficiency. SGSH removes the…”
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Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency by Luu, Amanda R., Wong, Cara, Agrawal, Vishal, Wise, Nathan, Handyside, Britta, Lo, Melanie J., Pacheco, Glenn, Felix, Jessica B., Giaramita, Alexander, d'Azzo, Alessandra, Vincelette, Jon, Bullens, Sherry, Bunting, Stuart, Christianson, Terri M., Hague, Charles M., LeBowitz, Jonathan H., Yogalingam, Gouri

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