A new step in understanding stem cell mobilization in patients with Fanconi anemia: A bridge to gene therapy

Authors: Diana, Jean‐sébastien, Manceau, Sandra, Leblanc, Thierry, Magnani, Alessandra, Magrin, Elisa, Bendavid, Matthieu, Couzin, Chloe, Joseph, Laure, Soulier, Jean, Cavazzana, Marina, Lefrère, Francois

Source: Transfusion. 62(1):165-172

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100139002225.0x000001&indx=1&recIds=ETOCvdc_100139002225.0x000001

Vascular access for optimal hematopoietic stem cell collection

Authors: Couzin, Chloé, Manceau, Sandra, Diana, Jean‐Sébastien, Joseph, Laure, Magnani, Alessandra, Magrin, Elisa, Amrane, Horiya, Dupont, Emilie, Raphalen, Jean‐Herlé, Sibon, David, Marcais, Ambroise, Suarez, Felipe, Cavazzana, Marina, Lefrère, François, Delville, Marianne

Source: Journal of clinical apheresis. 36(1):12-19

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100115430935.0x000001&indx=1&recIds=ETOCvdc_100115430935.0x000001

Procédé de préparation, contrôles de qualité et spécifications des immunosélections CD34+ : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC)

Alternate Title: CD34+ cell selection methods, quality controls and expected results: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) (English)

Authors: Calmels, Boris, Gautier, Éric, Magnani, Alessandra, Magrin, Élisa, Mamez, Anne-Claire, Vaissié, Alix, Yakoub-Agha, Ibrahim, Baudoux, Étienne ^*

Source: In Bulletin du Cancer December 2020 107(12) Supplement:S185-S192

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Authors: Six, Emmanuelle ^{1, 1, 2}, Guilloux, Agathe ³, Denis, Adeline ^{1, 2}, Lecoules, Arnaud ^{1, 2}, Magnani, Alessandra ⁴, Vilette, Romain ³, Male, Frances ⁵, Cagnard, Nicolas ^{2, 6}, Delville, Marianne ^{1, 2}, Magrin, Elisa ⁴, Caccavelli, Laure ⁴, Roudaut, Cécile ⁴, Plantier, Clemence ⁴, Sobrino, Steicy ^{1, 2}, Gregg, John ⁵, Nobles, Christopher L. ⁵, Everett, John K. ⁵, Hacein-Bey-Abina, Salima ^{7, 8}, Galy, Anne ^{9, 10}, Fischer, Alain ^{2, 11, 12, 13}, Thrasher, Adrian J. ¹⁴, André, Isabelle ^{1, 2}, Cavazzana, Marina ^{1, 2, 6, *}, Bushman, Frederic D. ^{2, 5, *}

Source: In Blood 9 April 2020 135(15):1219-1231

Bone Marrow Transplantation in Congenital Erythropoietic Porphyria: Sustained Efficacy but Unexpected Liver Dysfunction

Authors: Besnard, Caroline, Schmitt, Caroline, Galmiche-Rolland, Louise, Debray, Dominique, Fabre, Monique, Molina, Thierry, Gouya, Laurent, Ged, Cécile, Castelle, Martin, Cavazzana, Marina, Magrin, Elisa, Neven, Bénédicte, Moshous, Despina, Blanche, Stéphane, Frémond, Marie-Louise

Source: In Biology of Blood and Marrow Transplantation April 2020 26(4):704-711

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

Authors: Magrin, Elisa, Miccio, Annarita, Cavazzana, Marina

Source: In Blood 10 October 2019 134(15):1203-1213

Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma

Authors: Delville, Marianne, Touzot, Fabien, Couzin, Chloé, Hmitou, Isabelle, Djerroudi, Lounes, Ouedrani, Amani, Lefrère, François, Tuchman-Durand, Caroline, Mollet, Chloé, Fabreguettes, Jean-Roch, Ferry, Nicolas, Laganier, Laurent, Magnani, Alessandra, Magrin, Elisa, Jolaine, Valérie, Saez-Cirion, Asier, Wolstein, Orit, Symonds, Geoffrey, Frange, Pierre, Moins-Teisserenc, Hélène, Chaix-Baudier, Marie-Laure, Toubert, Antoine, Larghero, Jérôme, Parquet, Nathalie, Brignier, Anne C., Barré-Sinoussi, Françoise, Oksenhendler, Eric, Cavazzana, Marina

Source: In Molecular Therapy - Methods & Clinical Development 14 June 2019 13:303-309

Successful in utero stem cell transplantation in X-linked severe combined immunodeficiency

Authors: Magnani, Alessandra ^*, Jouannic, Jean-Marie ^*, Rosain, Jérémie, Gabrion, Aurélie, Touzot, Fabien, Roudaut, Cécile, Kracker, Sven, Mahlaoui, Nizar, Toubert, Antoine, Clave, Emmanuel, Macintyre, Elisabeth A., Radford-Weiss, Isabelle, Alcantara, Marion, Magrin, Elisa, Ternaux, Brigitte, Nisoy, Jennifer, Caccavelli, Laure, Darras, Anne-Marie, Picard, Capucine, Blanche, Stéphane, Cavazzana, Marina

Source: In Blood Advances 12 February 2019 3(3):237-241

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Authors: Weber, Leslie, Poletti, Valentina, Magrin, Elisa, Antoniani, Chiara, Martin, Samia, Bayard, Charles, Sadek, Hanem, Felix, Tristan, Meneghini, Vasco, Antoniou, Michael N., El-Nemer, Wassim, Mavilio, Fulvio, Cavazzana, Marina, Andre-Schmutz, Isabelle, Miccio, Annarita

Source: In Molecular Therapy - Methods & Clinical Development 21 September 2018 10:268-280

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Authors: Antoniani, Chiara ^*, Meneghini, Vasco ^*, Lattanzi, Annalisa, Felix, Tristan, Romano, Oriana, Magrin, Elisa, Weber, Leslie, Pavani, Giulia, El Hoss, Sara, Kurita, Ryo, Nakamura, Yukio, Cradick, Thomas J., Lundberg, Ante S., Porteus, Matthew, Amendola, Mario, El Nemer, Wassim, Cavazzana, Marina, Mavilio, Fulvio, Miccio, Annarita

Source: In Blood 26 April 2018 131(17):1960-1973

Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation

Authors: Coquerelle, Séverine, Ghardallou, Mariem, Rais, Setti, Taupin, Pierre, Touzot, Fabien, Boquet, Laure, Blanche, Stéphane, Benaouadi, Semir, Brice, Thomas, Tuchmann-Durand, Caroline, Ribeil, Jean Antoine, Magrin, Elisa, Lissillour, Etienne, Rochaix, Lise, Cavazzana, Marina, Durand-Zaleski, Isabelle

Source: Human gene therapy. 30(6):753-761

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Gene therapy of hemoglobinopathies: progress and future challenges

Authors: Ikawa, Yasuhiro, Miccio, Annarita, Magrin, Elisa, Kwiatkowski, Janet L, Rivella, Stefano, Cavazzana, Marina

Source: Human molecular genetics. 28(R1):R24-R30

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100096872280.0x000001&indx=1&recIds=ETOCvdc_100096872280.0x000001

The tyrosine phosphatase SHP-1 inhibits proliferation of activated hepatic stellate cells by impairing PDGF receptor signaling

Authors: Tibaldi, Elena, Zonta, Francesca, Bordin, Luciana, Magrin, Elisa, Gringeri, Enrico, Cillo, Umberto, Idotta, Giuseppe, Pagano, Mario Angelo, Brunati, Anna Maria

Source: In BBA - Molecular Cell Research February 2014 1843(2):288-298

Sickle Cell Disease: From Genetics to Curative Approaches.

Authors: Hardouin, Giulia^1,2,3 (AUTHOR) alice.corsia@gmail.com, Magrin, Elisa² (AUTHOR), Corsia, Alice³ (AUTHOR), Cavazzana, Marina^2,4,5 (AUTHOR), Miccio, Annarita¹ (AUTHOR), Semeraro, Michaela^5,6 (AUTHOR) michaela.semeraro@aphp.fr

Source: Annual Review of Genomics & Human Genetics. 2023, Vol. 24, p255-275. 21p.

Src tyrosine kinase preactivation is associated with platelet hypersensitivity in essential thrombocythemia and polycythemia vera

Authors: Randi, Maria Luigia ^*, Brunati, Anna Maria ^*, Scapin, Margherita, Frasson, Martina, Deana, Renzo, Magrin, Elisa, Fabris, Fabrizio, Donella-Deana, Arianna ^*

Source: In Blood 21 January 2010 115(3):667-676

Generation of adult human T-cell progenitors for immunotherapeutic applications

Authors: Simons, Laura, Ma, Kuiying, de Chappedelaine, Corinne, Moiranghtem, Ranjita Devi, Elkaim, Elodie, Olivré, Juliette, Susini, Sandrine, Appourchaux, Kevin, Reimann, Christian, Sadek, Hanem, Pellé, Olivier, Cagnard, Nicolas, Magrin, Elisa, Lagresle-Peyrou, Chantal, Taghon, Tom, Rausell, Antonio, Cavazzana, Marina, André-Schmutz, Isabelle

Source: journal of allergy and clinical immunology. 141(4):1491-1494.e4

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100056877451.0x000001&indx=1&recIds=ETOCvdc_100056877451.0x000001

Human Pro-T-Cell Manufacturing in Vitro Is a Safe Procedure for Hematopoietic Stem Cell Transplantation with Delayed T-Cell Reconstitution: Interim Results from 2 Different Clinical Trials

Authors: Magrin, Elisa, Diana, Jean-Sebastien, Castelle, Martin, Negre, Olivier, Simons, Laura, Aussel, Clotilde, Schwartz, Eden, Magnani, Alessandra, Baek, Jimni, Elie, Caroline, Maulet, Axelle, Mollet, Chloe, Girardot, Alice, Rosain, Jeremie, Picard, Capucine, Touzart, Aurore, Asnafi, Vahid, Forcade, Edouard, Huynh, Anne, Treluyer, jean-Marc, Andre, Isabelle, Moshous, Despina, Hermine, Olivier, Cavazzana, Marina

Source: In Blood 2 November 2023 142 Supplement 1:6868-6868

Preclinical Assessment for Gene Addition Strategy in Familial Hemophagocytic Lymphohistiocytosis Related to Munc 13-4 Deficiency

Authors: Lagresle-Peyrou, Chantal, Diana, Jean-Sebastien, Magrin, Elisa, Aussel, Clotilde, Sorel, Natael, Aslan, Yetki, Roudaut, Cécile, Schwartz, Eden, Gabrion, Aurélie, Tuchmann-Durand, Caroline, Lefrere, Francois, Galy, Anne, Moshous, Despina, Cavazzana, Marina

Source: In Blood 2 November 2023 142 Supplement 1:3633-3633

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

Authors: Magrin, Elisa, Semeraro, Michaela, Hebert, Nicolas, Joseph, Laure, Magnani, Alessandra, Chalumeau, Anne, Gabrion, Aurélie, Roudaut, Cécile, Marouene, Jouda, Lefrere, Francois, Diana, Jean-Sebastien, Denis, Adeline, Neven, Bénédicte, Funck-Brentano, Isabelle, Negre, Olivier, Renolleau, Sylvain, Brousse, Valentine, Kiger, Laurent, Touzot, Fabien, Poirot, Catherine, Bourget, Philippe, El Nemer, Wassim, Blanche, Stéphane, Tréluyer, Jean-Marc, Asmal, Mohammed, Walls, Courtney, Beuzard, Yves, Schmidt, Manfred, Hacein-Bey-Abina, Salima, Asnafi, Vahid, Guichard, Isabelle, Poirée, Maryline, Monpoux, Fabrice, Touraine, Philippe, Brouzes, Chantal, de Montalembert, Mariane, Payen, Emmanuel, Six, Emmanuelle, Ribeil, Jean-Antoine, Miccio, Annarita, Bartolucci, Pablo, Leboulch, Philippe, Cavazzana, Marina

Source: Nature Medicine; 20220101, Issue: Preprints p1-8, 8p

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