Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies

by Marina Cavazzana, Marina Cavazzana, Marina Cavazzana, Marina Cavazzana
Published 2017-03-01

Integration of retroviruses: a fine balance between efficiency and danger.

by Alain Fischer, Marina Cavazzana-Calvo
Published 2005-01-01

Concise Review: Boosting T‐Cell Reconstitution Following Allogeneic Transplantation—Current Concepts and Future Perspectives

by Laura Simons, Marina Cavazzana, Isabelle André
Published 2019-07-01

Alternative UNC13D Promoter Encodes a Functional Munc13-4 Isoform Predominantly Expressed in Lymphocytes and Platelets

by Donatella Galgano, Tayebeh Soheili, Matthias Voss, Lamberto Torralba-Raga, Bianca Tesi, Bianca Tesi, Frank Cichocki, Isabelle Andre, Isabelle Andre, Jens Rettig, Marina Cavazzana, Marina Cavazzana, Marina Cavazzana, Marina Cavazzana, Yenan Bryceson, Yenan Bryceson
Published 2020-06-01

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

by Frank J. T. Staal, Alessandro Aiuti, Alessandro Aiuti, Marina Cavazzana
Published 2019-10-01

An Evaluation of Three Ways of Communicating Carrier Status Results to the Parents of Children in a Neonatal Sickle Cell Screening Programme

by Christelle Rémus, Christelle Rémus, Christelle Rémus, Aurélie Stanislas, Aurélie Stanislas, Naïm Bouazza, Valérie Gauthereau, Michel Polak, Stéphane Blanche, Assa Niakaté, Eliane Gluckman, Jean-Marc Tréluyer, Arnold Munnich, Robert Girot, Robert Girot, Robert Girot, Marina Cavazzana, Marina Cavazzana
Published 2020-06-01

Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach

by Francesca Spanevello, Arianna Calistri, Claudia Del Vecchio, Barbara Mantelli, Chiara Frasson, Giuseppe Basso, Giorgio Palù, Marina Cavazzana, Cristina Parolin
Published 2016-01-01

A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells

by Marianne Delville, Tayebeh Soheili, Florence Bellier, Amandine Durand, Adeline Denis, Chantal Lagresle-Peyrou, Marina Cavazzana, Isabelle Andre-Schmutz, Emmanuelle Six
Published 2018-09-01

Two Monogenetic Disorders, Activated PI3-Kinase-δ Syndrome 2 and Smith–Magenis Syndrome, in One Patient: Case Report and a Literature Review of Neurodevelopmental Impact in Primary...

by Nidia Moreno-Corona, Nidia Moreno-Corona, Loïc Chentout, Loïc Chentout, Lucie Poggi, Lucie Poggi, Romane Thouenon, Romane Thouenon, Cecile Masson, Melanie Parisot, Lou Le Mouel, Capucine Picard, Capucine Picard, Capucine Picard, Isabelle André, Isabelle André, Marina Cavazzana, Marina Cavazzana, Laurence Perrin, Anne Durandy, Anne Durandy, Saba Azarnoush, Sven Kracker, Sven Kracker
Published 2021-06-01

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

by 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
Published 2019-12-01

Sustained remission after haploidentical bone marrow transplantation in a child with refractory systemic juvenile idiopathic arthritis

by Guillaume Morelle, Martin Castelle, Graziella Pinto, Sylvain Breton, Matthieu Bendavid, Charlotte Boussard, Richard Mouy, Brigitte Bader-Meunier, Michaela Semeraro, Albert Faye, Marina Cavazzana, Bénédicte Neven, Stéphane Blanche, Pierre Quartier, Despina Moshous
Published 2021-03-01

Real-time definition of non-randomness in the distribution of genomic events.

by Ulrich Abel, Annette Deichmann, Cynthia Bartholomae, Kerstin Schwarzwaelder, Hanno Glimm, Steven Howe, Adrian Thrasher, Alexandrine Garrigue, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Alain Fischer, Dirk Jaeger, Christof von Kalle, Manfred Schmidt
Published 2007-01-01

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

by Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N. Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio
Published 2018-09-01

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

by Charles C. Berry, Christopher Nobles, Emmanuelle Six, Yinghua Wu, Nirav Malani, Eric Sherman, Anatoly Dryga, John K. Everett, Frances Male, Aubrey Bailey, Kyle Bittinger, Mary J. Drake, Laure Caccavelli, Paul Bates, Salima Hacein-Bey-Abina, Marina Cavazzana, Frederic D. Bushman
Published 2017-03-01

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency

by Samantha L. Ginn, Claus V. Hallwirth, Sophia H.Y. Liao, Erdahl T. Teber, Jonathan W. Arthur, Jianmin Wu, Hong Ching Lee, Szun S. Tay, Min Hu, Roger R. Reddel, Matthew P. McCormack, Adrian J. Thrasher, Marina Cavazzana, Stephen I. Alexander, Ian E. Alexander
Published 2017-03-01

Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome.

by Mariane de Montalembert, Jean-Antoine Ribeil, Valentine Brousse, Agnes Guerci-Bresler, Aspasia Stamatoullas, Jean-Pierre Vannier, Cécile Dumesnil, Agnès Lahary, Mohamed Touati, Krimo Bouabdallah, Marina Cavazzana, Emmanuelle Chauzit, Amandine Baptiste, Thibaud Lefebvre, Hervé Puy, Caroline Elie, Zoubida Karim, Olivier Ernst, Christian Rose
Published 2017-01-01

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

by Eric Sherman, Christopher Nobles, Charles C. Berry, Emmanuelle Six, Yinghua Wu, Anatoly Dryga, Nirav Malani, Frances Male, Shantan Reddy, Aubrey Bailey, Kyle Bittinger, John K. Everett, Laure Caccavelli, Mary J. Drake, Paul Bates, Salima Hacein-Bey-Abina, Marina Cavazzana, Frederic D. Bushman
Published 2017-03-01

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency

by Erik L. Clarke, A. Jesse Connell, Emmanuelle Six, Nadia A. Kadry, Arwa A. Abbas, Young Hwang, John K. Everett, Casey E. Hofstaedter, Rebecca Marsh, Myriam Armant, Judith Kelsen, Luigi D. Notarangelo, Ronald G. Collman, Salima Hacein-Bey-Abina, Donald B. Kohn, Marina Cavazzana, Alain Fischer, David A. Williams, Sung-Yun Pai, Frederic D. Bushman
Published 2018-09-01

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

by Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A. Vloemans, Martijn Cordes, Axel Schambach, Michael Rothe, Dagmar Berghuis, Chantal Lagresle-Peyrou, Marina Cavazzana, Fang Zhang, Adrian J. Thrasher, Daniela Salvatori, Pauline Meij, Anna Villa, Jacques J.M. Van Dongen, Jaap-Jan Zwaginga, Mirjam van der Burg, H. Bobby Gaspar, Arjan Lankester, Frank J.T. Staal, Karin Pike-Overzet
Published 2020-06-01

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syn...

by Maria Elena Maccari, Maria Elena Maccari, Hassan Abolhassani, Hassan Abolhassani, Asghar Aghamohammadi, Alessandro Aiuti, Olga Aleinikova, Catherine Bangs, Safa Baris, Federica Barzaghi, Helen Baxendale, Matthew Buckland, Siobhan O. Burns, Caterina Cancrini, Caterina Cancrini, Andrew Cant, Pascal Cathébras, Marina Cavazzana, Marina Cavazzana, Marina Cavazzana, Anita Chandra, Anita Chandra, Francesca Conti, Francesca Conti, Tanya Coulter, Lisa A. Devlin, J. David M. Edgar, Saul Faust, Alain Fischer, Alain Fischer, Alain Fischer, Marina Garcia Prat, Lennart Hammarström, Maximilian Heeg, Maximilian Heeg, Stephen Jolles, Elif Karakoc-Aydiner, Gerhard Kindle, Ayca Kiykim, Dinakantha Kumararatne, Bodo Grimbacher, Hilary Longhurst, Nizar Mahlaoui, Nizar Mahlaoui, Tomas Milota, Fernando Moreira, Despina Moshous, Despina Moshous, Despina Moshous, Anna Mukhina, Olaf Neth, Benedicte Neven, Benedicte Neven, Benedicte Neven, Alexandra Nieters, Peter Olbrich, Ahmet Ozen, Jana Pachlopnik Schmid, Capucine Picard, Capucine Picard, Seraina Prader, William Rae, Janine Reichenbach, Stephan Rusch, Sinisa Savic, Alessia Scarselli, Alessia Scarselli, Raphael Scheible, Anna Sediva, Svetlana O. Sharapova, Anna Shcherbina, Mary Slatter, Pere Soler-Palacin, Aurelie Stanislas, Felipe Suarez, Francesca Tucci, Annette Uhlmann, Joris van Montfrans, Klaus Warnatz, Anthony Peter Williams, Phil Wood, Sven Kracker, Sven Kracker, Alison Mary Condliffe, Stephan Ehl, Stephan Ehl
Published 2018-03-01