RosaBacchettaMD

PubMed

• Identification of unstable regulatory and autoreactive effector T cells that are expanded in patients withmutations.
  Mavers, M., Šimon Borna, Esmond Lee, Jason Nideffer, Akshaya Ramachandran, Bing Wang, Jeanette Baker, Melissa Mavers, Uma Lakshmanan, Mansi Narula, Amy Kang-Hee Garret...> ;Science Translational Medicine. 2023 Dec 20
• 1 citations
  knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products.
  Yohei Sato, Abinaya Nathan, Suzette Shipp, John Fraser Wright, Keri Marie Tate, Prachi Wani, Maria-Grazia Roncarolo, Rosa Bacchetta> ;Molecular Therapy. Methods & Clinical Development. 2023 Dec 14
• 1 citations
  IPEX syndrome from diagnosis to cure, learning along the way.
  Rosa Bacchetta, Maria Grazia Roncarolo> ;The Journal of Allergy and Clinical Immunology. 2024 Mar 1
• FOXP3 deficiency, from the mechanisms of the disease to curative strategies.
  Simon Borna, Eric Meffre, Rosa Bacchetta> ;Immunological Reviews. 2024 Mar 1
• 2 citations
  FOXP3 TSDR Measurement Could Assist Variant Classification and Diagnosis of IPEX Syndrome.
  Rebecca C Wyatt, Sven Olek, Elisa De Franco, Bjoern Samans, Kashyap Patel, Jayne Houghton, Steffi Walter, Janika Schulze, Rosa Bacchetta, Andrew T Hattersley, Sarah E ...> ;Journal of Clinical Immunology. 2023 Apr 1
• Case report: Refractory Evans syndrome in two patients with spondyloenchondrodysplasia with immune dysregulation treated successfully with JAK1/JAK2 inhibition.
  Yael Gernez, Mansi Narula, Alma-Martina Cepika, Juanita Valdes Camacho, Elisabeth G Hoyte, Kirsten Mouradian, Bertil Glader, Deepika Singh, Bindu Sathi, Latha Rao, Ana...> ;Frontiers in Immunology. 2023 Jan 1
• Analyses of thymocyte commitment to regulatory T cell lineage in thymus of healthy subjects and patients with 22q11.2 deletion syndrome.
  Simon Borna, Beruh Dejene, Uma Lakshmanan, Janika Schulze, Kenneth Weinberg, Rosa Bacchetta> ;Frontiers in Immunology. 2023 Jan 1
• 7 citations
  Epigenetic and immunological indicators of IPEX disease in subjects with FOXP3 gene mutation.
  Mansi Narula, Uma Lakshmanan, Simon Borna, Janika J Schulze, Tyson H Holmes, Nicholas Harre, Matthew Kirkey, Akshaya Ramachandran, Veronica Maria Tagi, Federica Barzag...> ;The Journal of Allergy and Clinical Immunology. 2023 Jan 1
• 3 citations
  Rare immune diseases paving the road for genome editing-based precision medicine.
  Mara Pavel-Dinu, Simon Borna, Rosa Bacchetta> ;Frontiers in Genome Editing. 2023 Jan 1
• 7 citations
  Autoantibody discovery across monogenic, acquired, and COVID-19-associated autoimmunity with scalable PhIP-seq.
  Sara E Vazquez, Sabrina A Mann, Aaron Bodansky, Andrew F Kung, Zoe Quandt, Elise M N Ferré, Nils Landegren, Daniel Eriksson, Paul Bastard, Shen-Ying Zhang, Jamin Liu, ...> ;Elife. 2022 Oct 27
• 4 citations
  Design of experiments as a decision tool for cell therapy manufacturing.
  Esmond Lee, Devin Shah, Matthew Porteus, J Fraser Wright, Rosa Bacchetta> ;Cytotherapy. 2022 Jun 1
• 12 citations
  Towards gene therapy for IPEX syndrome.
  Simon Borna, Esmond Lee, Yohei Sato, Rosa Bacchetta> ;European Journal of Immunology. 2022 May 1
• Editorial: IPEX 2020: An Expanding Disease Spectrum and Novel Precision Therapies.
  Rosa Bacchetta, Talal Chatila> ;Frontiers in Pediatrics. 2022 Jan 1
• 5 citations
  Pre-clinical development and molecular characterization of an engineered type 1 regulatory T-cell product suitable for immunotherapy.
  Jeffrey Liu, Ping Chen, Molly Javier Uyeda, Brandon Cieniewicz, Ece Canan Sayitoglu, Benjamin Craig Thomas, Yohei Sato, Rosa Bacchetta, Alma-Martina Cepika, Maria Graz...> ;Cytotherapy. 2021 Nov 1
• 30 citations
  Alloantigen-specific type 1 regulatory T cells suppress through CTLA-4 and PD-1 pathways and persist long-term in patients.
  Pauline P. Chen, Alma-Martina Cepika, Rajni Agarwal-Hashmi, Gopin Saini, Molly Javier Uyeda, David M. Louis, Brandon Cieniewicz, Mansi Narula, Laura C. Amaya Hernandez...> ;Science Translational Medicine. 2021 Oct 27
• 14 citations
  Co-Expression of FOXP3FL and FOXP3Δ2 Isoforms Is Required for Optimal Treg-Like Cell Phenotypes and Suppressive Function.
  Yohei Sato, Jessica Liu, Esmond Lee, Rhonda Perriman, Maria Grazia Roncarolo, Rosa Bacchetta> ;Frontiers in Immunology. 2021 Oct 19
• 8 citations
  BHLHE40 Regulates IL-10 and IFN-γ Production in T Cells but Does Not Interfere With Human Type 1 Regulatory T Cell Differentiation.
  Molly Javier Uyeda, Robert Arthur Freeborn, Brandon Cieniewicz, Rosa Romano, Ping Pauline Chen, Jeffrey Liu, Benjamin Craig Thomas, Esmond Lee, Alma-Martina Cepika, Ro...> ;Frontiers in Immunology. 2021 Jul 7
• 5 citations
  Thymic origins of autoimmunity-lessons from inborn errors of immunity.
  Rosa Bacchetta, Kenneth I. Weinberg> ;Seminars in Immunopathology. 2021 Feb 2
• 10 citations
  Engineered type 1 regulatory T cells designed for clinical use kill primary pediatric acute myeloid leukemia cells
  Brandon Cieniewicz, Molly Javier Uyeda, Ping Pauline Chen, Ece Canan Sayitoglu, Jeffrey Liu, Grazia Andolfi, Katharine Greenthal, Alice Bertaina, Silvia Gregori, Rosa ...> ;Haematologica. 2020 Sep 28
• 111 citations
  Human inborn errors of immunity: An expanding universe.
  Luigi D. Notarangelo, Rosa Bacchetta, Jean-Laurent Casanova, Helen C. Su> ;Science Immunology. 2020 Jul 10
• 41 citations
  Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey
  Alice Y. Chan, Jennifer W. Leiding, Xuerong Liu, Brent R. Logan, Lauri Burroughs, Eric J. Allenspach, Suzanne Skoda-Smith, Gulbu Uzel, Luigi D. Notarangelo, Mary Slatt...> ;Frontiers in Immunology. 2020 Feb 21
• 25 citations
  Human-engineered Treg-like cells suppress FOXP3-deficient T cells but preserve adaptive immune responses in vivo
  Yohei Sato, Laura Passerini, Brian D. Piening, Molly Javier Uyeda, Marianne Goodwin, Silvia Gregori, Michael Snyder, Alice Bertaina, Maria Grazia Roncarolo, Rosa Bacch...> ;Clinical & Translational Immunology. 2020 Jan 1
• 66 citations
  Severe autoinflammation in 4 patients with C-terminal variants in cell division control protein 42 homolog (CDC42) successfully treated with IL-1β inhibition.
  Yael Gernez, Adriana Almeida de Jesus, Hanouf Alsaleem, Claudia Macaubas, A. Roy, Daniel J. Lovell, Karthik A. Jagadeesh, Sara Alehashemi, Laura K. Erdman, Michael Gri...> ;The Journal of Allergy and Clinical Immunology. 2019 Jul 2
• 8 citations
  The autoimmune targets in IPEX are dominated by gut epithelial proteins.
  Daniel Eriksson, Rosa Bacchetta, Hörður Ingi Gunnarsson, Alice Y. Chan, Federica Barzaghi, Stephan Ehl, Åsa Hallgren, Frédéric Van Gool, Fabian Sardh, Christina Lundqv...> ;The Journal of Allergy and Clinical Immunology. 2019 Jul 1
• 23 citations
  Case Study: Mechanism for Increased Follicular Helper T Cell Development in Activated PI3K Delta Syndrome.
  Timothy J. Thauland, Laurence Pellerin, Robert S. Ohgami, Rosa Bacchetta, Manish J. Butte> ;Frontiers in Immunology. 2019 Apr 12
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