| No | Title of Publication | DOI | Authors | Title of Journal |
| 1 | Refining strategies to translate genome editing to the clinic | 10.1038/nm.43
13 |
Tatjana I Cornu, Claudio Mussolino, Toni Cathomen | Nature Medicine |
| 2 | Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements | 10.1016/j.ymth
e.2018.10.008 |
Annalisa Lattanzi, Vasco Meneghini, Giulia Pavani, Fatima Amor, Sophie Ramadier, Tristan Felix, Chiara Antoniani, Cecile Masson, Olivier Alibeu, Ciaran Lee, Matthew H. Porteus, Gang Bao, Mario Amendola, Fulvio Mavilio, Annarita Miccio | Molecular Therapy |
| 3 | Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes | 10.1016/j.biom
aterials.2016.0 4.019 |
Verena Labenski, Julia D. Suerth, Elke Barczak, Dirk Heckl, Camille Levy, Ornellie Bernadin, Emmanuelle Charpentier, David A. Williams, Boris Fehse, Els Verhoeyen, Axel Schambach | Biomaterials |
| 4 | Generation of adult human Tcell progenitors for immunotherape utic applications | 10.1016/j.jaci.2
017.10.034 |
Laura Simons, Kuiying Ma, Corinne de Chappedelaine, Ranjita Devi Moiranghtem, Elodie Elkaim, Juliette Olivré, Sandrine Susini, Kevin Appourchaux, Christian Reimann, Hanem Sadek, Olivier Pellé, Nicolas Cagnard, Elisa Magrin, Chantal Lagresle-Peyrou, Tom Taghon, Antonio Rausell, Marina Cavazzana, Isabelle André | Journal of Allergy and Clinical Immunology |
| 5 | Efficacy of lentivirusmediated gene therapy in an Omenn syndrome recombinationactivating gene 2 mouse model is not hindered by inflammation and immune dysregulation | 10.1016/j.jaci.2
017.11.015 |
Valentina Capo, Maria Carmina Castiello, Elena Fontana, Sara Penna, Marita Bosticardo, Elena Draghici, Luigi P. Poliani, Lucia Sergi Sergi, Rosita Rigoni, Barbara Cassani, Monica Zanussi, Paola Carrera, Paolo Uva, Kerry Dobbs, Nicolò Sacchetti, Luigi D. Notarangelo, Niek P. van Til, Gerard Wagemaker, Anna Villa | Journal of Allergy and Clinical Immunology |
| 6 | Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration | 10.1089/hgtb.2
017.073 |
Maike Stahlhut, Axel Schambach, Olga S. Kustikova | Human Gene Therapy Methods |
| 7 | Therapeutic genome editing with engineered nucleases | 10.5482/HAMO
-16-09-0035 |
S. A. Haas, V. Dettmer, T. Cathomen | Hämostaseologie |
| 8 | Viral and Synthetic RNA Vector Technologies and Applications | 10.1038/mt.201
6.143 |
Juliane W Schott, Michael Morgan, Melanie Galla, Axel Schambach | Molecular
Therapy
|
| 9 | An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries | 10.1016/j.biom
aterials.2017.0 5.032 |
Felix F. Adams, Dirk Heckl, Thomas Hoffmann, Steven R. Talbot, Arnold Kloos, Felicitas Thol, Michael Heuser, Johannes
Zuber, Axel Schambach, Adrian Schwarzer |
Biomaterials |
| 10 | How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID) | 10.1007/s1087
5-017-0373-y |
Donald B. Kohn, H. Bobby Gaspar
|
Journal of Clinical Immunology |
| 11 | Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency | 10.3389/fimmu.
2016.00314
|
Kathryn V. Whitmore, Hubert B. Gaspar | Frontiers in Immunology |
| 12 | Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations | 10.1007/s1087
5-017-0433-3 |
Kathryn L. Bradford, Federico A. Moretti, Denise A. Carbonaro-Sarracino, Hubert B. Gaspar, Donald B. Kohn | Journal of Clinical Immunology |
| 13 | Gene Therapy Approaches to Immunodeficiency | 10.1016/j.hoc.2
017.05.003 |
Sujal Ghosh, H. Bobby Gaspar | Hematology/Oncology Clinics of North America |
| 14 | Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency | 10.1016/j.omtm
.2018.03.002 |
Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio | Molecular Therapy Methods & Clinical Development |
| 15 | Treating Immunodeficiency through HSC Gene Therapy | 10.1016/j.molm
ed.2016.02.002 |
Claire Booth, H. Bobby Gaspar, Adrian J. Thrasher | Trends in Molecular Medicine |
| 16 | Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency | 10.1016/j.jaci.2
018.08.024 |
Donald B. Kohn, Michael S. Hershfield, Jennifer M. Puck, Alessandro Aiuti, Annaliesse Blincoe, H. Bobby Gaspar, Luigi D. Notarangelo, Eyal Grunebaum | Journal of Allergy and Clinical Immunology |
