The clinical landscape for AAV gene therapies
Kuzmin, Dmitry A, Shutova, Maria V, Johnston, Natalie R, Smith, Owen P, Fedorin, Vasily V, Kukushkin, Yury S, van der Loo, Johannes C M, Johnstone, Elaine C
Published in Nature reviews. Drug discovery (01.03.2021)
Published in Nature reviews. Drug discovery (01.03.2021)
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Journal Article
Familial pulmonary alveolar proteinosis caused by mutations in CSF2RA
Suzuki, Takuji, Sakagami, Takuro, Rubin, Bruce K, Nogee, Lawrence M, Wood, Robert E, Zimmerman, Sarah L, Smolarek, Teresa, Dishop, Megan K, Wert, Susan E, Whitsett, Jeffrey A, Grabowski, Gregory, Carey, Brenna C, Stevens, Carrie, van der Loo, Johannes C M, Trapnell, Bruce C
Published in The Journal of experimental medicine (24.11.2008)
Published in The Journal of experimental medicine (24.11.2008)
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Journal Article
Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization
Liu, Xueyuan, Jean-Gilles, Riffard, Baginski, Julia, Cai, Christina, Yan, Ruilan, Zhang, Lili, Lance, Kevin, van der Loo, Johannes C.M., Davidson, Beverly L.
Published in Molecular therapy. Methods & clinical development (12.09.2024)
Published in Molecular therapy. Methods & clinical development (12.09.2024)
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Journal Article
Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency
Nasimuzzaman, Md, Lynn, Danielle, Ernst, Rebecca, Beuerlein, Michele, Smith, Richard H, Shrestha, Archana, Cross, Scott, Link, Kevin, Lutzko, Carolyn, Nordling, Diana, Russell, David W, Larochelle, Andre, Malik, Punam, Van der Loo, Johannes C M
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Journal Article
Nonobese Diabetic/Severe Combined Immunodeficiency (NOD/SCID) Mouse as a Model System to Study the Engraftment and Mobilization of Human Peripheral Blood Stem Cells
van der Loo, Johannes C.M., Hanenberg, Helmut, Cooper, Ryan J., Luo, F.-Y., Lazaridis, Emmanuel N., Williams, David A.
Published in Blood (01.10.1998)
Published in Blood (01.10.1998)
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Journal Article
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
George, Lindsey A, Sullivan, Spencer K, Giermasz, Adam, Rasko, John E.J, Samelson-Jones, Benjamin J, Ducore, Jonathan, Cuker, Adam, Sullivan, Lisa M, Majumdar, Suvankar, Teitel, Jerome, McGuinn, Catherine E, Ragni, Margaret V, Luk, Alvin Y, Hui, Daniel, Wright, J. Fraser, Chen, Yifeng, Liu, Yun, Wachtel, Katie, Winters, Angela, Tiefenbacher, Stefan, Arruda, Valder R, van der Loo, Johannes C.M, Zelenaia, Olga, Takefman, Daniel, Carr, Marcus E, Couto, Linda B, Anguela, Xavier M, High, Katherine A
Published in The New England journal of medicine (07.12.2017)
Published in The New England journal of medicine (07.12.2017)
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Journal Article
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
Hacein-Bey-Abina, Salima, Pai, Sung-Yun, Gaspar, H. Bobby, Armant, Myriam, Berry, Charles C, Blanche, Stephane, Bleesing, Jack, Blondeau, Johanna, de Boer, Helen, Buckland, Karen F, Caccavelli, Laure, Cros, Guilhem, De Oliveira, Satiro, Fernández, Karen S, Guo, Dongjing, Harris, Chad E, Hopkins, Gregory, Lehmann, Leslie E, Lim, Annick, London, Wendy B, van der Loo, Johannes C.M, Malani, Nirav, Male, Frances, Malik, Punam, Marinovic, M. Angélica, McNicol, Anne-Marie, Moshous, Despina, Neven, Benedicte, Oleastro, Matías, Picard, Capucine, Ritz, Jerome, Rivat, Christine, Schambach, Axel, Shaw, Kit L, Sherman, Eric A, Silberstein, Leslie E, Six, Emmanuelle, Touzot, Fabien, Tsytsykova, Alla, Xu-Bayford, Jinhua, Baum, Christopher, Bushman, Frederic D, Fischer, Alain, Kohn, Donald B, Filipovich, Alexandra H, Notarangelo, Luigi D, Cavazzana, Marina, Williams, David A, Thrasher, Adrian J
Published in The New England journal of medicine (09.10.2014)
Published in The New England journal of medicine (09.10.2014)
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Journal Article
Process Development for the Production and Purification of Adeno-Associated Virus (AAV)2 Vector using Baculovirus-Insect Cell Culture System
Nasimuzzaman, Md, Villaveces, Sophia, van der Loo, Johannes C M, Alla, Sivani
Published in Journal of visualized experiments (13.01.2022)
Published in Journal of visualized experiments (13.01.2022)
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Journal Article
Production and Purification of Baculovirus for Gene Therapy Application
Nasimuzzaman, Md, van der Loo, Johannes C.M., Malik, Punam
Published in Journal of visualized experiments (09.04.2018)
Published in Journal of visualized experiments (09.04.2018)
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Journal Article
Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of Leukocyte Adhesion Deficiency Type 1
Smith, Richard H, Bloomer, Hanan, Fink, Danielle, Keyvanfar, Keyvan, Nasimuzzaman, Md, Sancheznieto, Fátima, Dutta, Roop, Guenther Bui, Kacey, Alvarado, Luigi J, Bauer, Jr, Thomas R, Hickstein, Dennis D, Russell, David W, Malik, Punam, van der Loo, Johannes C M, Highfill, Steven L, Kuhns, Douglas B, Pirooznia, Mehdi, Larochelle, Andre
Published in Human gene therapy (01.12.2022)
Published in Human gene therapy (01.12.2022)
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Journal Article
Foamy Virus Backbone Has Insulator Properties Which Remarkably Reduce Its Genotoxicity Potential
Goodman, Michael, Arumugam, Paritha, Pillis, Devin, Lynn, Danielle, Van der Loo, Johannes C.M., Keddache, Mehdi, Bauer, Thomas R., Hickstein, Dennis D., Russell, David W, Malik, Punam
Published in Blood (02.12.2016)
Published in Blood (02.12.2016)
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Journal Article
Production and Purification of Baculovirus for Gene Therapy Application
Nasimuzzaman, Md, van der Loo, Johannes C M, Malik, Punam
Published in Journal of visualized experiments (09.04.2018)
Published in Journal of visualized experiments (09.04.2018)
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Journal Article
Selecting a Cell Engineering Methodology During Cell Therapy Product Development
Timmins, Lauren M., Burr, Alexandra M., Carroll, Kristina, Keefe, Robert, Teryek, Matthew, Cantolupo, Louis J., van der Loo, Johannes C. M., Heathman, Thomas R.J., Gormley, Adam J, Smith, David, Parekkadan, Biju
Published in Cell Transplantation (2021)
Published in Cell Transplantation (2021)
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Book Review
Journal Article
Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype
Malik, Punam, Grimley, Michael, Quinn, Charles T., Shova, Amy, Courtney, Little, Lutzko, Carolyn, Kalfa, Theodosia A., Niss, Omar, Mehta, Parinda A, Chandra, Sharat, Grassman, Elke, Van der Loo, Johannes C.M., Witting, Scott, Nordling, Diana, Shreshta, Archana, Felker, Sydney, Terrell, Catherine, Reeves, Lilith, Pillis, Devin, Anastacia, Loberg, Bushman, Frederic D, Knight-Madden, Jennifer, Kalinyak, Karen, Davies, Stella M., Asnani, Monika
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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Journal Article
461. Scale-Up and Manufacturing of High-Titer Foamy Virus Vector Containing Human CD18 for the Treatment of Leukocyte Adhesion Deficiency
Nasimuzzaman, Md, Lynn, Danielle, Ernst, Rebecca, Beuerlein, Michele, Cross, Scott, Link, Kevin, Lutzko, Carolyn, Nordling, Diana, Russell, David, Malik, Punam, Van der Loo, Johannes C.M.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Journal Article