AAV‐mediated delivery of an anti‐BACE1 VHH alleviates pathology in an Alzheimer's disease model
Marino, Marika, Zhou, Lujia, Rincon, Melvin Y, Callaerts‐Vegh, Zsuzsanna, Verhaert, Jens, Wahis, Jérôme, Creemers, Eline, Yshii, Lidia, Wierda, Keimpe, Saito, Takashi, Marneffe, Catherine, Voytyuk, Iryna, Wouters, Yessica, Dewilde, Maarten, Duqué, Sandra I, Vincke, Cécile, Levites, Yona, Golde, Todd E, Saido, Takaomi C, Muyldermans, Serge, Liston, Adrian, De Strooper, Bart, Holt, Matthew G
Published in EMBO molecular medicine (07.04.2022)
Published in EMBO molecular medicine (07.04.2022)
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An immunoaffinity-based method for isolating ultrapure adult astrocytes based on ATP1B2 targeting by the ACSA-2 antibody
Batiuk, Mykhailo Y., de Vin, Filip, Duqué, Sandra I., Li, Chen, Saito, Takashi, Saido, Takaomi, Fiers, Mark, Belgard, T. Grant, Holt, Matthew G.
Published in The Journal of biological chemistry (26.05.2017)
Published in The Journal of biological chemistry (26.05.2017)
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Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector
Rincon, Melvin Y., de Vin, Filip, Duqué, Sandra I., Fripont, Shelly, Castaldo, Stephanie A., Bouhuijzen-Wenger, Jessica, Holt, Matthew G.
Published in Gene therapy (01.04.2018)
Published in Gene therapy (01.04.2018)
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Mild SMN missense alleles are only functional in the presence of SMN2 in mammals
Iyer, Chitra C, Corlett, Kaitlyn M, Massoni-Laporte, Aurélie, Duque, Sandra I, Madabusi, Narasimhan, Tisdale, Sarah, McGovern, Vicki L, Le, Thanh T, Zaworski, Phillip G, Arnold, W David, Pellizzoni, Livio, Burghes, Arthur H M
Published in Human molecular genetics (01.10.2018)
Published in Human molecular genetics (01.10.2018)
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A large animal model of spinal muscular atrophy and correction of phenotype
Duque, Sandra I., Arnold, W. David, Odermatt, Philipp, Li, Xiaohui, Porensky, Paul N., Schmelzer, Leah, Meyer, Kathrin, Kolb, Stephen J., Schümperli, Daniel, Kaspar, Brian K., Burghes, Arthur H. M.
Published in Annals of neurology (01.03.2015)
Published in Annals of neurology (01.03.2015)
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Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null mice
McGovern, Vicki L, Kray, Kaitlyn M, Arnold, W David, Duque, Sandra I, Iyer, Chitra C, Massoni-Laporte, Aurélie, Workman, Eileen, Patel, Aalapi, Battle, Daniel J, Burghes, Arthur H M
Published in Human molecular genetics (01.11.2020)
Published in Human molecular genetics (01.11.2020)
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SMN Blood Levels in a Porcine Model of Spinal Muscular Atrophy
Iyer, Chitra, Wang, Xueqian, Renusch, Samantha R., Duque, Sandra I., Wehr, Allison M., Mo, Xiaokui-Molly, McGovern, Vicki L., Arnold, W. David, Burghes, Arthur H.M., Kolb, Stephen J.
Published in Journal of neuromuscular diseases (2017)
Published in Journal of neuromuscular diseases (2017)
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Journal Article
Mild SMN missense alleles are only functional in the presence of SMN2 in mammals
Iyer, Chitra C, Corlett, Kaitlyn M, Massoni-Laporte, Aurélie, Duque, Sandra I, Madabusi, Narasimhan, Tisdale, Sarah, McGovern, Vicki L, Le, Thanh T, Zaworski, Phillip G, Arnold, W David, Pellizzoni, Livio, Burghes, Arthur H M
Published in Human molecular genetics (01.10.2018)
Published in Human molecular genetics (01.10.2018)
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Journal Article
AAV mediated delivery of a novel anti-BACE1 VHH reduces Abeta in an Alzheimer's disease mouse model
Rincon, Melvin, Zhou, Lujia, Marneffe, Catherine, Voytyuk, Iryna, Wouters, Yessica, Dewilde, Maarten, Duqué, Sandra I, Vincke, Cécile, Levites, Yona, Golde, Todd E, Muyldermans, Serge, De Strooper, Bart, Holt, Matthew G
Published in bioRxiv (13.07.2019)
Published in bioRxiv (13.07.2019)
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