Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease von Jeyakumar, Jey M., Kia, Azadeh, Tam, Lawrence C. S., McIntosh, Jenny, Spiewak, Justyna, Mills, Kevin, Heywood, Wendy, Chisari, Elisa, Castaldo, Noemi, Verhoef, Daniël, Hosseini, Paniz, Kalcheva, Petya, Cocita, Clement, Miranda, Carlos J., Canavese, Miriam, Khinder, Jaminder, Rosales, Cecilia, Hughes, Derralynn, Sheridan, Rose, Corbau, Romuald, Nathwani, Amit

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Gene targeting as a therapeutic avenue in diseases mediated by the complement alternative pathway von Dreismann, Anna K, Hallam, Thomas M, Tam, Lawrence CS, Nguyen, Calvin V, Hughes, Jane P, Ellis, Scott, Harris, Claire L

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Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma von O'Callaghan, Jeffrey, Crosbie, Darragh E, Cassidy, Paul S, Sherwood, Joseph M, Flügel-Koch, Cassandra, Lütjen-Drecoll, Elke, Humphries, Marian M, Reina-Torres, Ester, Wallace, Deborah, Kiang, Anna-Sophia, Campbell, Matthew, Stamer, W Daniel, Overby, Darryl R, O'Brien, Colm, Tam, Lawrence C S, Humphries, Peter

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A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement von BOWNE, Sara J, HUMPHRIES, Marian M, FULTON, Robert S, SODERGREN, Erica J, ALLMAN, Denis, MILLINGTON-WARD, Sophia, PALFI, Arpad, MCKEE, Alex, BLANTON, Susan H, SLIFER, Susan, KONIDARI, Loanna, FARRAR, G. Jane, SULLIVAN, Lori S, DAIGER, Stephen P, HUMPHRIES, Peter, KENNA, Paul F, TAM, Lawrence Cs, KIANG, Anna S, CAMPBELL, Matthew, WEINSTOCK, George M, KOBOLDT, Daniel C, LI DING

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experimental platform for systemic drug delivery to the retina von Campbell, Matthew, Nguyen, Anh T.H, Kiang, Anna-Sophia, Tam, Lawrence C.S, Gobbo, Oliviero L, Kerskens, Christian, Dhubhghaill, Sorcha Ni, Humphries, Marian M, Farrar, G.-Jane, Kenna, Paul F, Humphries, Peter

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Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90) von Tam, Lawrence C.S., Kiang, Anna-Sophia, Campbell, Matthew, Keaney, James, Jane Farrar, G., Humphries, Marian M., Kenna, Paul F., Humphries, Pete

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Age-related changes in eye morphology and aqueous humor dynamics in DBA/2J mice using contrast-enhanced ocular MRI von Crosbie, Darragh E., Keaney, James, Tam, Lawrence C.S., Daniel Stamer, W., Campbell, Matthew, Humphries, Peter

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Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10) von Tam, Lawrence C.S., Kiang, Anna-Sophia, Kennan, Avril, Kenna, Paul F., Chadderton, Naomi, Ader, Marius, Palfi, Arpad, Aherne, Aileen, Ayuso, Carmen, Campbell, Matthew, Reynolds, Alison, McKee, Alex, Humphries, Marian M., Farrar, G. Jane, Humphries, Pete

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Enhancement of Outflow Facility in the Murine Eye by Targeting Selected Tight-Junctions of Schlemm’s Canal Endothelia von Tam, Lawrence C. S., Reina-Torres, Ester, Sherwood, Joseph M., Cassidy, Paul S., Crosbie, Darragh E., Lütjen-Drecoll, Elke, Flügel-Koch, Cassandra, Perkumas, Kristin, Humphries, Marian M., Kiang, Anna-Sophia, O’Callaghan, Jeffrey, Callanan, John J., Read, A. Thomas, Ethier, C. Ross, O’Brien, Colm, Lawrence, Matthew, Campbell, Matthew, Stamer, W. Daniel, Overby, Darryl R., Humphries, Pete

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A Genomic DNA Reporter Screen Identifies Squalene Synthase Inhibitors That Act Cooperatively with Statins to Upregulate the Low-Density Lipoprotein Receptor von Kerr, Alastair G, Tam, Lawrence C S, Hale, Ashley B, Cioroch, Milena, Douglas, Gillian, Agkatsev, Sarina, Hibbitt, Olivia, Mason, Joseph, Holt-Martyn, James, Bataille, Carole J R, Wynne, Graham M, Channon, Keith M, Russell, Angela J, Wade-Martins, Richard

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Episomal Nonviral Gene Therapy Vectors Slow Progression of Atherosclerosis in a Model of Familial Hypercholesterolemia von Kerr, Alastair G, Tam, Lawrence CS, Hale, Ashley B, Cioroch, Milena, Douglas, Gillian, Channon, Keith M, Wade-Martins, Richard

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C1q enhances cone photoreceptor survival in a mouse model of autosomal recessive retinitis pigmentosa von HUMPHRIES, Marian M, KENNA, Paul F, CAMPBELL, Matthew, TAM, Lawrence Cs, NGUYEN, Anh Th, JANE FARRAR, G, BOTTO, Marina, SOPHIA KIANG, Anna, HUMPHRIES, Peter

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Systemic low‐molecular weight drug delivery to pre‐selected neuronal regions von Campbell, Matthew, Humphries, Marian M., Kiang, Anna‐Sophia, Nguyen, Anh T. H., Gobbo, Oliviero L., Tam, Lawrence C. S., Suzuki, Mayu, Hanrahan, Finnian, Ozaki, Ema, Farrar, G.‐Jane, Kenna, Paul F., Humphries, Peter

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Toward a Gene Therapy for Dominant Disease: Validation of an RNA Interference-Based Mutation-Independent Approach von Kiang, Anna-Sophia, Palfi, Arpad, Ader, Marius, Kenna, Paul F, Millington-Ward, Sophia, Clark, Gerry, Kennan, Avril, O'reilly, Mary, Tam, Lawrence C T, Aherne, Aileen, McNally, Niamh, Humphries, Pete, Farrar, G Jane

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α‐Fluorotricarbonyl Derivatives as Versatile Fluorinated Building Blocks: Synthesis of Fluoroacetophenone, Fluoroketo Ester and Fluoropyran‐4‐one Derivatives von Harsanyi, Antal, Lückener, Anne, Pasztor, Hedvig, Yilmaz, Zahide, Tam, Lawrence, Yufit, Dmitry S., Sandford, Graham

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A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement von Bowne, Sara J, Humphries, Marian M, Sullivan, Lori S, Kenna, Paul F, Tam, Lawrence Cs, Kiang, Anna S, Campbell, Matthew, Weinstock, George M, Koboldt, Daniel C, Ding, Li, Fulton, Robert S, Sodergren, Erica J, Allman, Denis, Millington-ward, Sophia, Palfi, Arpad, Mckee, Alex, Blanton, Susan H, Slifer, Susan, Konidari, Ioanna, Farrar, G Jane, Daiger, Stephen P, Humphries, Peter

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How Relevant Are Library and Information Science Curricula outside Their Geographic Domain? von Tam, Lawrence Wai-hong, Harvey, Ross, Mills, John

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Managing change: libraries and information services in the digital age von Tam, Lawrence W.H., Robertson, Averil C.

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Abstract 228: A Novel Combinatorial Non-viral Vector to Treat Familial Hypercholesterolaemia von Kerr, Alastair G, Tam, Lawrence, Cioroch, Milena, Hale, Ashley, Douglas, Gillian, Channon, Keith, Wade-Martins, Richard

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19. Using a Novel Combinatorial Non-Viral Vector and Small Molecules to Treat Familial Hypercholesterolaemia (FH) von Kerr, Alastair G., Tam, Lawrence, Cioroch, Milena, Hale, Ashley, Douglas, Gillian, Mason, Joe, Bataille, Carole, Wynne, Graham, Russell, Angela, Channon, Keith, Wade-Martins, Richard

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