No Title of Publication DOI Authors Title of Journal
1 Refining strategies to translate genome editing to the clinic 10.1038/nm.43


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


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



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


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


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


Maike Stahlhut, Axel Schambach, Olga S. Kustikova Human Gene Therapy Methods
7 Therapeutic genome editing with engineered nucleases 10.5482/HAMO


S. A. Haas, V. Dettmer, T. Cathomen Hämostaseologie
8 Viral and Synthetic RNA Vector Technologies and Applications 10.1038/mt.201


Juliane W Schott, Michael Morgan, Melanie Galla, Axel Schambach Molecular



9 An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries 10.1016/j.biom



Felix F. Adams, Dirk Heckl, Thomas Hoffmann, Steven R. Talbot, Arnold Kloos, Felicitas Thol, Michael Heuser, Johannes

Zuber, Axel Schambach, Adrian Schwarzer

10 How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID) 10.1007/s1087


Donald B. Kohn, H. Bobby Gaspar


Journal of Clinical Immunology
11 Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency 10.3389/fimmu.



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


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


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


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


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


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