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Michael J. Lenardo
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Journal Articles

A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome

Clinical Trials & Observations
V. Koneti Rao, Sharon Webster, Anna Šedivá, Alessandro Plebani, Catharina Schuetz, Anna Shcherbina, Niall Conlon, Tanya Coulter, Virgil A. Dalm, Antonino Trizzino, Yulia Zharankova, Elaine Kulm, Julia Körholz, Vassilios Lougaris, Yulia Rodina, Kath Radford, Jason Bradt, Klaus Kucher, Anurag Relan, Steven M. Holland, Michael J. Lenardo, Gulbu Uzel
Journal: Blood
Blood (2023) 141 (9): 971–983.
https://doi.org/10.1182/blood.2022018546
Published: 2023
Includes: Supplemental data
Journal Articles

CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Julie Brault, Taylor Liu, Ezekiel Bello, Siyuan Liu, Colin L. Sweeney, Ronald J. Meis, Sherry Koontz, Cristina Corsino, Uimook Choi, Guillaume Vayssiere, Marita Bosticardo, Kennichi Dowdell, Cicera R. Lazzarotto, Aaron B. Clark, Luigi D. Notarangelo, Juan C. Ravell, Michael J. Lenardo, Benjamin P. Kleinstiver, Shengdar Q. Tsai, Xiaolin Wu, Gary A. Dahl, Harry L. Malech, Suk See De Ravin
Journal: Blood
Blood (2021) 138 (26): 2768–2780.
https://doi.org/10.1182/blood.2021011192
Published: 2021
Includes: Supplemental data
Journal Articles

Extended clinical and immunological phenotype and transplant outcome in CD27 and CD70 deficiency

Sujal Ghosh, Sevgi Köstel Bal, Emily S. J. Edwards, Bethany Pillay, Raúl Jiménez Heredia, Funda Erol Cipe, Geetha Rao, Elisabeth Salzer, Samaneh Zoghi, Hassan Abolhassani, Tooba Momen, Emma Gostick, David A. Price, Yu Zhang, Andrew J. Oler, Claudia Gonzaga-Jauregui, Baran Erman, Ayse Metin, Inci Ilhan, Sule Haskologlu, Candan Islamoglu, Kubra Baskin, Serdar Ceylaner, Ebru Yilmaz, Ekrem Unal, Musa Karakukcu, Dagmar Berghuis, Theresa Cole, Aditya K. Gupta, Fabian Hauck, Hubert Kogler, Andy I. M. Hoepelman, Safa Baris, Elif Karakoc-Aydiner, Ahmet Ozen, Leo Kager, Dirk Holzinger, Michael Paulussen, Renate Krüger, Roland Meisel, Prasad T. Oommen, Emma Morris, Benedicte Neven, Austen Worth, Joris van Montfrans, Pieter L. A. Fraaij, Sharon Choo, Figen Dogu, E. Graham Davies, Siobhan Burns, Gregor Dückers, Ruy Perez Becker, Horst von Bernuth, Sylvain Latour, Maura Faraci, Marco Gattorno, Helen C. Su, Qiang Pan-Hammarström, Lennart Hammarström, Michael J. Lenardo, Cindy S. Ma, Tim Niehues, Asghar Aghamohammadi, Nima Rezaei, Aydan Ikinciogullari, Stuart G. Tangye, Arjan C. Lankester, Kaan Boztug
Journal: Blood
Blood (2020) 136 (23): 2638–2655.
https://doi.org/10.1182/blood.2020006738
Published: 2020
Includes: Supplemental data
Journal Articles

Effective “activated PI3Kδ syndrome”–targeted therapy with the PI3Kδ inhibitor leniolisib

Clinical Trials & Observations
V. Koneti Rao, Sharon Webster, Virgil A. S. H. Dalm, Anna Šedivá, P. Martin van Hagen, Steven Holland, Sergio D. Rosenzweig, Andreas D. Christ, Birgitte Sloth, Maciej Cabanski, Aniket D. Joshi, Stefan de Buck, Julie Doucet, Danilo Guerini, Christoph Kalis, Ilona Pylvaenaeinen, Nicolas Soldermann, Anuj Kashyap, Gulbu Uzel, Michael J. Lenardo, Dhavalkumar D. Patel, Carrie L. Lucas, Christoph Burkhart
Journal: Blood
Blood (2017) 130 (21): 2307–2316.
https://doi.org/10.1182/blood-2017-08-801191
Published: 2017
Includes: Supplemental data
Journal Articles

CHAI and LATAIE: new genetic diseases of CTLA-4 checkpoint insufficiency

Clinical Trials & Observations
Bernice Lo, Jill M. Fritz, Helen C. Su, Gulbu Uzel, Michael B. Jordan, Michael J. Lenardo
Journal: Blood
Blood (2016) 128 (8): 1037–1042.
https://doi.org/10.1182/blood-2016-04-712612
Published: 2016
Journal Articles

XMEN disease: a new primary immunodeficiency affecting Mg 2+ regulation of immunity against Epstein-Barr virus

Feng-Yen Li, Benjamin Chaigne-Delalande, Helen Su, Gulbu Uzel, Helen Matthews, Michael J. Lenardo
Journal: Blood
Blood (2014) 123 (14): 2148–2152.
https://doi.org/10.1182/blood-2013-11-538686
Published: 2014
Journal Articles

Natural history of autoimmune lymphoproliferative syndrome associated with FAS gene mutations

Susan Price, Pamela A. Shaw, Amy Seitz, Gyan Joshi, Joie Davis, Julie E. Niemela, Katie Perkins, Ronald L. Hornung, Les Folio, Philip S. Rosenberg, Jennifer M. Puck, Amy P. Hsu, Bernice Lo, Stefania Pittaluga, Elaine S. Jaffe, Thomas A. Fleisher, V. Koneti Rao, Michael J. Lenardo
Journal: Blood
Blood (2014) 123 (13): 1989–1999.
https://doi.org/10.1182/blood-2013-10-535393
Published: 2014
Includes: Supplemental data
Journal Articles

Revised diagnostic criteria and classification for the autoimmune lymphoproliferative syndrome (ALPS): report from the 2009 NIH International Workshop

e-blood
Joao B. Oliveira, Jack J. Bleesing, Umberto Dianzani, Thomas A. Fleisher, Elaine S. Jaffe, Michael J. Lenardo, Frederic Rieux-Laucat, Richard M. Siegel, Helen C. Su, David T. Teachey, V. Koneti Rao
Journal: Blood
Blood (2010) 116 (14): e35–e40.
https://doi.org/10.1182/blood-2010-04-280347
Published: 2010
Includes: Supplemental data
Journal Articles

Haploinsufficiency, rather than the effect of an excessive production of soluble CD95 (CD95ΔTM), is the basis for ALPS Ia in a family with duplicated 3′ splice site AG in CD95 intron 5 on one allele

Clinical Trials & Observations
Joachim Roesler, Jose-Maria Izquierdo, Martin Ryser, Angela Rösen-Wolff, Manfred Gahr, Juan Valcarcel, Michael J. Lenardo, Lixin Zheng
Journal: Blood
Blood (2005) 106 (5): 1652–1659.
https://doi.org/10.1182/blood-2004-08-3104
Published: 2005
Includes: Supplemental data
Journal Articles

Immunophenotypic profiles in families with autoimmune lymphoproliferative syndrome

Clinical Trials & Observations
Jack J. H. Bleesing, Margaret R. Brown, Stephen E. Straus, Janet K. Dale, Richard M. Siegel, Michele Johnson, Michael J. Lenardo, Jennifer M. Puck, Thomas A. Fleisher
Journal: Blood
Blood (2001) 98 (8): 2466–2473.
https://doi.org/10.1182/blood.V98.8.2466
Published: 2001
Journal Articles

The development of lymphomas in families with autoimmune lymphoproliferative syndrome with germline Fas mutations and defective lymphocyte apoptosis

Stephen E. Straus, Elaine S. Jaffe, Jennifer M. Puck, Janet K. Dale, Keith B. Elkon, Angela Rösen-Wolff, Anke M. J. Peters, Michael C. Sneller, Claire W. Hallahan, Jin Wang, Roxanne E. Fischer, Christine M. Jackson, Albert Y. Lin, Caroline Bäumler, Elke Siegert, Alexander Marx, Akshay K. Vaishnaw, Tamara Grodzicky, Thomas A. Fleisher, Michael J. Lenardo
Journal: Blood
Blood (2001) 98 (1): 194–200.
https://doi.org/10.1182/blood.V98.1.194
Published: 2001
Journal Articles

Clinical, Immunologic, and Genetic Features of an Autoimmune Lymphoproliferative Syndrome Associated With Abnormal Lymphocyte Apoptosis

Michael C. Sneller, Jin Wang, Janet K. Dale, Warren Strober, Lindsay A. Middelton, Youngnim Choi, Thomas A. Fleisher, Megan S. Lim, Elaine S. Jaffe, Jennifer M. Puck, Michael J. Lenardo, Stephen E. Straus
Journal: Blood
Blood (1997) 89 (4): 1341–1348.
https://doi.org/10.1182/blood.V89.4.1341
Published: 1997