Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

Por Cavazzana, Marina, Six, Emmanuelle, Lagresle-Peyrou, Chantal, André-Schmutz, Isabelle, Hacein-Bey-Abina, Salima
Publicado no Hum Gene Ther (2016)

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

Por Cavazzana-Calvo, Marina, Fischer, Alain, Bushman, Frederic D., Payen, Emmanuel, Hacein-Bey-Abina, Salima, Leboulch, Philippe
Publicado em 2011

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Por Wang, Gary P., Berry, Charles C., Malani, Nirav, Leboulch, Philippe, Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Bushman, Frederic D.
Publicado em 2010

Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

Por van der Loo, JCM, Swaney, WP, Grassman, E, Terwilliger, A, Higashimoto, T, Schambach, A, Hacein-Bey-Abina, S, Nordling, DL, Cavazzana-Calvo, M, Thrasher, AJ, Williams, DA, Reeves, L, Malik, P
Publicado em 2012

Assessing the effect of genetic markers on drug immunogenicity from a mechanistic model-based approach

Por Julianne Duhazé, Miguel Caubet, Signe Hässler, Delphine Bachelet, Matthieu Allez, Florian Deisenhammer, Anna Fogdell-Hahn, Aude Gleizes, Salima Hacein-Bey-Abina, Xavier Mariette, Marc Pallardy, Philippe Broët, on behalf of the ABIRISK Consortium
Publicado em 2020-03-01

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

Por Wang, Gary P., Garrigue, Alexandrine, Ciuffi, Angela, Ronen, Keshet, Leipzig, Jeremy, Berry, Charles, Lagresle-Peyrou, Chantal, Benjelloun, Fatine, Hacein-Bey-Abina, Salima, Fischer, Alain, Cavazzana-Calvo, Marina, Bushman, Frederic D.
Publicado em 2008

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

Por Sabine Charrier, Chantal Lagresle-Peyrou, Valentina Poletti, Michael Rothe, Grégory Cédrone, Bernard Gjata, Fulvio Mavilio, Alain Fischer, Axel Schambach, Jean-Pierre de Villartay, Marina Cavazzana, Salima Hacein-Bey-Abina, Anne Galy
Publicado em 2019-12-01

Real-Time Definition of Non-Randomness in the Distribution of Genomic Events

Por Abel, Ulrich, Deichmann, Annette, Bartholomae, Cynthia, Schwarzwaelder, Kerstin, Glimm, Hanno, Howe, Steven, Thrasher, Adrian, Garrigue, Alexandrine, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Fischer, Alain, Jaeger, Dirk, von Kalle, Christof, Schmidt, Manfred
Publicado em 2007

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Por Zhou, Sheng, Mody, Disha, DeRavin, Suk See, Hauer, Julia, Lu, Taihe, Ma, Zhijun, Hacein-Bey Abina, Salima, Gray, John T., Greene, Michael R., Cavazzana-Calvo, Marina, Malech, Harry L., Sorrentino, Brian P.
Publicado em 2010

A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

Por Brady, Troy, Roth, Shoshannah L., Malani, Nirav, Wang, Gary P., Berry, Charles C., Leboulch, Philippe, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Papapetrou, Eirini P., Sadelain, Michel, Savilahti, Harri, Bushman, Frederic D.
Publicado em 2011

Activation-induced cytidine deaminase prevents pro-B cell acute lymphoblastic leukemia by functioning as a negative regulator in Rag1 deficient pro-B cells

Por Auer, Franziska, Ingenhag, Deborah, Pinkert, Stefan, Kracker, Sven, Hacein-Bey-Abina, Salima, Cavazzana, Marina, Gombert, Michael, Martin-Lorenzo, Alberto, Lin, Min-Hui, Vicente-Dueñas, Carolina, Sánchez-García, Isidro, Borkhardt, Arndt, Hauer, Julia
Publicado no Oncotarget (2017)

Loss of p19Arf in a Rag1(−/−) B-cell precursor population initiates acute B-lymphoblastic leukemia

Por Hauer, Julia, Mullighan, Charles, Morillon, Estelle, Wang, Gary, Bruneau, Julie, Brousse, Nicole, Lelorc'h, Marc, Romana, Serge, Boudil, Amine, Tiedau, Daniela, Kracker, Sven, Bushmann, Frederic D., Borkhardt, Arndt, Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina
Publicado em 2011

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

Por Berry, Charles C., Nobles, Christopher, Six, Emmanuelle, Wu, Yinghua, Malani, Nirav, Sherman, Eric, Dryga, Anatoly, Everett, John K., Male, Frances, Bailey, Aubrey, Bittinger, Kyle, Drake, Mary J., Caccavelli, Laure, Bates, Paul, Hacein-Bey-Abina, Salima, Cavazzana, Marina, Bushman, Frederic D.
Publicado no Mol Ther Methods Clin Dev (2016)

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes

Por Sherman, Eric, Nobles, Christopher, Berry, Charles C., Six, Emmanuelle, Wu, Yinghua, Dryga, Anatoly, Malani, Nirav, Male, Frances, Reddy, Shantan, Bailey, Aubrey, Bittinger, Kyle, Everett, John K., Caccavelli, Laure, Drake, Mary J., Bates, Paul, Hacein-Bey-Abina, Salima, Cavazzana, Marina, Bushman, Frederic D.
Publicado no Mol Ther Methods Clin Dev (2016)

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Por Hacein-Bey-Abina, Salima, Hauer, Julia, Lim, Annick, Picard, Capucine, Wang, Gary P., Berry, Charles C., Martinache, Chantal, Rieux-Laucat, Frédéric, Latour, Sylvain, Belohradsky, Bernd H., Leiva, Lily, Sorensen, Ricardo, Debré, Marianne, Casanova, Jean Laurent, Blanche, Stephane, Durandy, Anne, Bushman, Frederic D., Fischer, Alain, Cavazzana-Calvo, Marina
Publicado em 2010

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Por Hacein-Bey-Abina, Salima, Garrigue, Alexandrine, Wang, Gary P., Soulier, Jean, Lim, Annick, Morillon, Estelle, Clappier, Emmanuelle, Caccavelli, Laure, Delabesse, Eric, Beldjord, Kheira, Asnafi, Vahid, MacIntyre, Elizabeth, Dal Cortivo, Liliane, Radford, Isabelle, Brousse, Nicole, Sigaux, François, Moshous, Despina, Hauer, Julia, Borkhardt, Arndt, Belohradsky, Bernd H., Wintergerst, Uwe, Velez, Maria C., Leiva, Lily, Sorensen, Ricardo, Wulffraat, Nicolas, Blanche, Stéphane, Bushman, Frederic D., Fischer, Alain, Cavazzana-Calvo, Marina
Publicado em 2008

Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy

Por Deichmann, Annette, Brugman, Martijn H, Bartholomae, Cynthia C, Schwarzwaelder, Kerstin, Verstegen, Monique MA, Howe, Steven J, Arens, Anne, Ott, Marion G, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Fischer, Alain, Paruzynski, Anna, Gabriel, Richard, Glimm, Hanno, Abel, Ulrich, Cattoglio, Claudia, Mavilio, Fulvio, Cassani, Barbara, Aiuti, Alessandro, Dunbar, Cynthia E, Baum, Christopher, Gaspar, H Bobby, Thrasher, Adrian J, von Kalle, Christof, Schmidt, Manfred, Wagemaker, Gerard
Publicado em 2011

Site- and allele-specific polycomb dysregulation in T-cell leukaemia

Por Navarro, Jean-Marc, Touzart, Aurore, Pradel, Lydie C., Loosveld, Marie, Koubi, Myriam, Fenouil, Romain, Le Noir, Sandrine, Maqbool, Muhammad Ahmad, Morgado, Ester, Gregoire, Claude, Jaeger, Sebastien, Mamessier, Emilie, Pignon, Charles, Hacein-Bey-Abina, Salima, Malissen, Bernard, Gut, Marta, Gut, Ivo G., Dombret, Hervé, Macintyre, Elizabeth A., Howe, Steven J., Gaspar, H. Bobby, Thrasher, Adrian J., Ifrah, Norbert, Payet-Bornet, Dominique, Duprez, Estelle, Andrau, Jean-Christophe, Asnafi, Vahid, Nadel, Bertrand
Publicado no Nat Commun (2015)

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

Por Deichmann, Annette, Hacein-Bey-Abina, Salima, Schmidt, Manfred, Garrigue, Alexandrine, Brugman, Martijn H., Hu, Jingqiong, Glimm, Hanno, Gyapay, Gabor, Prum, Bernard, Fraser, Christopher C., Fischer, Nicolas, Schwarzwaelder, Kerstin, Siegler, Maria-Luise, de Ridder, Dick, Pike-Overzet, Karin, Howe, Steven J., Thrasher, Adrian J., Wagemaker, Gerard, Abel, Ulrich, Staal, Frank J.T., Delabesse, Eric, Villeval, Jean-Luc, Aronow, Bruce, Hue, Christophe, Prinz, Claudia, Wissler, Manuela, Klanke, Chuck, Weissenbach, Jean, Alexander, Ian, Fischer, Alain, von Kalle, Christof, Cavazzana-Calvo, Marina
Publicado em 2007

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Por Cavazzana-Calvo, Marina, Payen, Emmanuel, Negre, Olivier, Wang, Gary, Hehir, Kathleen, Fusil, Floriane, Down, Julian, Denaro, Maria, Brady, Troy, Westerman, Karen, Cavallesco, Resy, Gillet-Legrand, Beatrix, Caccavelli, Laure, Sgarra, Riccardo, Maouche-Chrétien, Leila, Bernaudin, Françoise, Girot, Robert, Dorazio, Ronald, Mulder, Geert-Jan, Polack, Axel, Bank, Arthur, Soulier, Jean, Larghero, Jérôme, Kabbara, Nabil, Dalle, Bruno, Gourmel, Bernard, Socie, Gérard, Chrétien, Stany, Cartier, Nathalie, Aubourg, Patrick, Fischer, Alain, Cornetta, Kenneth, Galacteros, Frédéric, Beuzard, Yves, Gluckman, Eliane, Bushman, Frederick, Hacein-Bey-Abina, Salima, Leboulch, Philippe
Publicado em 2010