The extended clinical phenotype of 64 patients with dedicator of cytokinesis 8 deficiency

Authors: Engelhardt, Karin R., Gertz, Michael E., Keles, Sevgi, Schäffer, Alejandro A., Sigmund, Elena C., Glocker, Cristina, Saghafi, Shiva, Pourpak, Zahra, Ceja, Ruben, Sassi, Atfa, Graham, Laura E., Massaad, Michel J., Mellouli, Fethi, Ben-Mustapha, Imen, Khemiri, Monia, Kilic, Sara Sebnem, Etzioni, Amos, Freeman, Alexandra F., Thiel, Jens, Schulze, Ilka, Al-Herz, Waleed, Metin, Ayse, Sanal, Özden, Tezcan, Ilhan, Yeganeh, Mehdi, Niehues, Tim, Dueckers, Gregor, Weinspach, Sebastian, Patiroglu, Turkan, Unal, Ekrem, Dasouki, Majed, Yilmaz, Mustafa, Genel, Ferah, Aytekin, Caner, Kutukculer, Necil, Somer, Ayper, Kilic, Mehmet, Reisli, Ismail, Camcioglu, Yildiz, Gennery, Andrew R., Cant, Andrew J., Jones, Alison, Gaspar, Bobby H., Arkwright, Peter D., Pietrogrande, Maria C., Baz, Zeina, Al-Tamemi, Salem, Lougaris, Vassilios, Lefranc, Gerard, Megarbane, Andre, Boutros, Jeannette, Galal, Nermeen, Bejaoui, Mohamed, Barbouche, Mohamed-Ridha, Geha, Raif S., Chatila, Talal A., Grimbacher, Bodo

Source: In The Journal of Allergy and Clinical Immunology August 2015 136(2):402-412

Tandem mass spectrometry, but not T-cell receptor excision circle analysis, identifies newborns with late-onset adenosine deaminase deficiency

Authors: la Marca, Giancarlo, Canessa, Clementina, Giocaliere, Elisa, Romano, Francesca, Duse, Marzia, Malvagia, Sabrina, Lippi, Francesca, Funghini, Silvia, Bianchi, Leila, Della Bona, Maria Luisa, Valleriani, Claudia, Ombrone, Daniela, Moriondo, Maria, Villanelli, Fabio, Speckmann, Carsten, Adams, Stuart, Gaspar, Bobby H., Hershfield, Michael, Santisteban, Ines, Fairbanks, Lynette, Ragusa, Giovanni, Resti, Massimo, de Martino, Maurizio, Guerrini, Renzo, Azzari, Chiara

Source: In The Journal of Allergy and Clinical Immunology June 2013 131(6):1604-1610

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Authors: Magnani, Alessandra, Semeraro, Michaela, ADAM, Frédéric, Booth, Claire, Dupre, Loic, Morris, Emma C, Gabrion, Aurélie, Roudaut, Cecile, Borgel, Delphine, Toubert, Antoine, Clave, Emmanuel, Abdo, Chrystelle, Gorochov, Guy, Petermann, Rachel, Guiot, Mélanie, Miyara, Makoto, Moshous, Despina, Magrin, Elisa, Denis, Adeline, Suarez, Felipe, Lagresle-Peyrou, Chantal, Doto, Aoife, Everett, John K., Trinquand, Amélie, Guisset, Marianne, Xu-Bayford, Jin Hua, Hacein-Bey-Abina, Salima, Kauskot, Alexandre, Elfeky, Reem, Rivat, Christine, Abbas, Sarah, Gaspar, Bobby H, Macintyre, Elizabeth A., Picard, Capucine, Bushman, Frederic D., Galy, Anne, Fischer, Alain, Six, Emmanuelle, Thrasher, Adrian J., Cavazzana, Marina

Source: In Blood 23 November 2021 138 Supplement 1:2934-2934

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Authors: Izotova, Natalia ^*, Rivat, Christine ^*, Baricordi, Cristina ^*, Blanco-Alvarez, Elena ^*, Pellin, Danilo ^*, Watt, Eleanor ^*, Gkazi, Soragia Athina ^*, Adams, Stuart ^*, Gilmour, Kimberly ^*, Bayford, Jinhua Xu ^*, Booth, Claire ^*, Gaspar, Bobby H ^*, Thrasher, Adrian J. ^*, Biasco, Luca

Source: In Blood 5 November 2020 136 Supplement 1:50-50

HSCT for DOCK8 Deficiency - an International Study on 74 Patients

Authors: Aydin, Susanne, Freeman, Alexandra F., Su, Helen, Hickstein, Dennis, Pai, Sung-Yun, Geha, Raif, Chatila, Talal, Arnaout, Rand K., Al-Mousa, Hamoud, Al-Dhekri, Hasan, Renner, Ellen D., Belohradsky, Bernd H., Notheis, Gundula, Aydin, Roland, Gaspar, Bobby H., Al-Herz, Waleed, Gennery, Andrew, Slatter, Mary, Metin, Ayse, Isik, Pamir, Azik, Fatih, Ikinciogullari, Aydan, Haskologlu, Sule, Dogu, Figen, Kumar, Ashish, Ifversen, Marianne, Schuster, Friedhelm, Woessmann, Wilhelm, Bredius, Robbert, Barlogis, Vincent, Kainulainen, Leena, Picard, Capucine, Neven, Benedicte, Shereck, Evan, Schulz, Ansgar, Dueckers, Gregor, Van Montfrans, Joris, Matthes-Martin, Susanne, Keles, Sevgi, Ochs, Hans, Albert, Michael H.

Source: In Biology of Blood and Marrow Transplantation March 2016 22(3) Supplement:S103-S104

243. Pre-Clinical Development of Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency

Authors: Poletti, Valentina, Charrier, Sabine, Martin, Samia, Moiani, Arianna, Sagrère, Céline, Ferrand, Sandrine, Hebben, Matthias, Zhang, Fang, Buckland, Karen, Rothe, Michael, Schambach, Alex, Gaspar, Bobby H., Thrasher, Adrian, Mavilio, Fulvio

Source: In Molecular Therapy May 2015 23 Supplement 1:S95-S95

237. Genetic Therapy for Perforin Deficiency Associated Hemophagocytic Lymphohistiocytosis Requires High Level Expression of the Perforin Gene for Adequate Correction

Authors: Tiwari, Swati, Hontz, Adrianne, Terrell, Catherine, Arumugam, Paritha, Carmo, Marlene, Gaspar, Bobby H., Risma, Kimberly, Jordan, Michael, Malik, Punam

Source: In Molecular Therapy May 2015 23 Supplement 1:S93-S93

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