Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
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
Published in Gene therapy (01.06.2023)
Published in Gene therapy (01.06.2023)
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Journal Article
Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma
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
Published in Human molecular genetics (01.04.2017)
Published in Human molecular genetics (01.04.2017)
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Journal Article
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement
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
Published in European journal of human genetics : EJHG (01.10.2011)
Published in European journal of human genetics : EJHG (01.10.2011)
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Journal Article
experimental platform for systemic drug delivery to the retina
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
Published in Proceedings of the National Academy of Sciences - PNAS (20.10.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (20.10.2009)
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Journal Article
Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90)
Tam, Lawrence C.S., Kiang, Anna-Sophia, Campbell, Matthew, Keaney, James, Jane Farrar, G., Humphries, Marian M., Kenna, Paul F., Humphries, Pete
Published in Human molecular genetics (15.11.2010)
Published in Human molecular genetics (15.11.2010)
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Journal Article
Age-related changes in eye morphology and aqueous humor dynamics in DBA/2J mice using contrast-enhanced ocular MRI
Crosbie, Darragh E., Keaney, James, Tam, Lawrence C.S., Daniel Stamer, W., Campbell, Matthew, Humphries, Peter
Published in Magnetic resonance imaging (01.06.2019)
Published in Magnetic resonance imaging (01.06.2019)
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Journal Article
Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10)
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
Published in Human molecular genetics (15.07.2008)
Published in Human molecular genetics (15.07.2008)
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Journal Article
Enhancement of Outflow Facility in the Murine Eye by Targeting Selected Tight-Junctions of Schlemm’s Canal Endothelia
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
Published in Journal of the Statistical and Social Inquiry Society of Ireland (16.01.2017)
Published in Journal of the Statistical and Social Inquiry Society of Ireland (16.01.2017)
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Journal Article
A Genomic DNA Reporter Screen Identifies Squalene Synthase Inhibitors That Act Cooperatively with Statins to Upregulate the Low-Density Lipoprotein Receptor
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
Published in The Journal of pharmacology and experimental therapeutics (01.06.2017)
Published in The Journal of pharmacology and experimental therapeutics (01.06.2017)
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Journal Article
Episomal Nonviral Gene Therapy Vectors Slow Progression of Atherosclerosis in a Model of Familial Hypercholesterolemia
Kerr, Alastair G, Tam, Lawrence CS, Hale, Ashley B, Cioroch, Milena, Douglas, Gillian, Channon, Keith M, Wade-Martins, Richard
Published in Molecular therapy. Nucleic acids (08.11.2016)
Published in Molecular therapy. Nucleic acids (08.11.2016)
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Journal Article
C1q enhances cone photoreceptor survival in a mouse model of autosomal recessive retinitis pigmentosa
HUMPHRIES, Marian M, KENNA, Paul F, CAMPBELL, Matthew, TAM, Lawrence Cs, NGUYEN, Anh Th, JANE FARRAR, G, BOTTO, Marina, SOPHIA KIANG, Anna, HUMPHRIES, Peter
Published in European journal of human genetics : EJHG (01.01.2012)
Published in European journal of human genetics : EJHG (01.01.2012)
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Journal Article
Systemic low‐molecular weight drug delivery to pre‐selected neuronal regions
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
Published in EMBO molecular medicine (01.04.2011)
Published in EMBO molecular medicine (01.04.2011)
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Journal Article
Toward a Gene Therapy for Dominant Disease: Validation of an RNA Interference-Based Mutation-Independent Approach
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
Published in Molecular therapy (01.09.2005)
Published in Molecular therapy (01.09.2005)
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Journal Article
Protection of photoreceptors in a mouse model of RP10
Tam, Lawrence C S, Kiang, Anna-Sophia, Chadderton, Naomi, Kenna, Paul F, Campbell, Matthew, Humphries, Marian M, Farrar, G Jane, Humphries, Pete
Published in Advances in experimental medicine and biology (2010)
Published in Advances in experimental medicine and biology (2010)
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Journal Article
Reversible and size-selective opening of the inner Blood-Retina barrier: a novel therapeutic strategy
Campbell, Matthew, Nguyen, Anh Thi Hong, Kiang, Anna-Sophia, Tam, Lawrence, Kenna, Paul F, Dhubhghaill, Sorcha Ni, Humphries, Marian, Farrar, G Jane, Humphries, Peter
Published in Advances in experimental medicine and biology (01.01.2010)
Published in Advances in experimental medicine and biology (01.01.2010)
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Journal Article
Protein misfolding and potential therapeutic treatments in inherited retinopathies
Tam, Lawrence C S, Kiang, Anna-Sophia, Campbell, Matthew, Keaney, James, Farrar, G Jane, Humphries, Marian M, Kenna, Paul F, Humphries, Pete
Published in Advances in experimental medicine and biology (2012)
Published in Advances in experimental medicine and biology (2012)
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Journal Article
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement
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
Published in European journal of human genetics : EJHG (01.10.2011)
Published in European journal of human genetics : EJHG (01.10.2011)
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Journal Article
How Relevant Are Library and Information Science Curricula outside Their Geographic Domain?
Tam, Lawrence Wai-hong, Harvey, Ross, Mills, John
Published in Education for information (01.01.2007)
Published in Education for information (01.01.2007)
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Journal Article
On further development of barrier modulation as a technique for systemic ocular drug delivery
Hanrahan, Finnian, Campbell, Matthew, Nguyen, Anh T, Suzuki, Mayu, Kiang, Anna-Sophia, Tam, Lawrence C, Gobbo, Oliviero L, Dhubhghaill, Sorcha Ní, Humphries, Marian M, Kenna, Paul F, Humphries, Pete
Published in Advances in experimental medicine and biology (2012)
Published in Advances in experimental medicine and biology (2012)
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Journal Article