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2. CRISPR Medicine: Advance, Progress and Challenges

Author

Ureña-Bailén, Guillermo, primary, Antony, Justin S., additional, Hou, Yujuan, additional, Raju, Janani, additional, Lamsfus-Calle, Andres, additional, Daniel-Moreno, Alberto, additional, Handgretinger, Rupert, additional, and Mezger, Markus, additional

Published
2021
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5. Automated Good Manufacturing Practice-Compatible CRISPR-Cas9 Editing of Hematopoietic Stem and Progenitor Cells for Clinical Treatment of β-Hemoglobinopathies

Author

Ureña-Bailén, Guillermo, primary, Block, Milena, additional, Grandi, Tommaso, additional, Aivazidou, Faidra, additional, Quednau, Jona, additional, Krenz, Dariusz, additional, Daniel-Moreno, Alberto, additional, Lamsfus-Calle, Andrés, additional, Epting, Thomas, additional, Handgretinger, Rupert, additional, Wild, Stefan, additional, and Mezger, Markus, additional

Published
2023
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7. Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing

Author

Hou, Yujuan, primary, Ureña-Bailén, Guillermo, additional, Mohammadian Gol, Tahereh, additional, Gratz, Paul Gerhard, additional, Gratz, Hans Peter, additional, Roig-Merino, Alicia, additional, Antony, Justin S., additional, Lamsfus-Calle, Andrés, additional, Daniel-Moreno, Alberto, additional, Handgretinger, Rupert, additional, and Mezger, Markus, additional

Published
2022
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8. Preclinical Evaluation of CRISPR-Edited CAR-NK-92 Cells for Off-the-Shelf Treatment of AML and B-ALL

Author

Ureña-Bailén, Guillermo, primary, Dobrowolski, Jérôme-Maurice, additional, Hou, Yujuan, additional, Dirlam, Alicia, additional, Roig-Merino, Alicia, additional, Schleicher, Sabine, additional, Atar, Daniel, additional, Seitz, Christian, additional, Feucht, Judith, additional, Antony, Justin S., additional, Mohammadian Gol, Tahereh, additional, Handgretinger, Rupert, additional, and Mezger, Markus, additional

Published
2022
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9. A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy

Author

Antony, Justin S., primary, Daniel-Moreno, Alberto, additional, Lamsfus-Calle, Andrés, additional, Raju, Janani, additional, Kaftancioglu, Merve, additional, Ureña-Bailén, Guillermo, additional, Rottenberger, Jennifer, additional, Hou, Yujuan, additional, Santhanakumaran, Vidiyaah, additional, Lee, Jun-Hoe, additional, Heumos, Lukas, additional, Böhringer, Judith, additional, Krägeloh-Mann, Ingeborg, additional, Handgretinger, Rupert, additional, and Mezger, Markus, additional

Published
2022
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10. Somatic Reversion of a Novel IL2RG Mutation Resulting in Atypical X-Linked Combined Immunodeficiency

Author

Hou, Yujuan, primary, Gratz, Hans Peter, additional, Ureña-Bailén, Guillermo, additional, Gratz, Paul G., additional, Schilbach-Stückle, Karin, additional, Renno, Tina, additional, Güngör, Derya, additional, Mader, Daniel A., additional, Malenke, Elke, additional, Antony, Justin S., additional, Handgretinger, Rupert, additional, and Mezger, Markus, additional

Published
2021
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12. Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates

Author

Lamsfus-Calle, Andrés, primary, Daniel-Moreno, Alberto, additional, Ureña-Bailén, Guillermo, additional, Rottenberger, Jennifer, additional, Raju, Janani, additional, Epting, Thomas, additional, Marciano, Sabina, additional, Heumos, Lukas, additional, Baskaran, Praveen, additional, S Antony, Justin, additional, Handgretinger, Rupert, additional, and Mezger, Markus, additional

Published
2021
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14. CRISPR/Cas9 technology: towards a new generation of improved CAR-T cells for anticancer therapies.

Author

Ureña-Bailén, Guillermo, Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Raju, Janani, Schlegel, Patrick, Seitz, Christian, Atar, Daniel, Antony, Justin S, Handgretinger, Rupert, and Mezger, Markus

Subjects
*CELLULAR therapy, *CHIMERIC antigen receptors, *GENOME editing, *GENE therapy, *TECHNOLOGICAL innovations, *CYTOTOXIC T cells
Abstract

Chimeric antigen receptor (CAR)-modified T cells have raised among other immunotherapies for cancer treatment, being implemented against B-cell malignancies. Despite the promising outcomes of this innovative technology, CAR-T cells are not exempt from limitations that must yet to be overcome in order to provide reliable and more efficient treatments against other types of cancer. The purpose of this review is to shed light on the field of CAR-T cell gene editing for therapy universalization and further enhancement of antitumor function. Several studies have proven that the disruption of certain key genes is essential to boost immunosuppressive resistance, prevention of fratricide, and clinical safety. Due to its unparalleled simplicity, feasibility to edit multiple gene targets simultaneously, and affordability, CRISPR/CRISPR-associated protein 9 system has been proposed in different clinical trials for such CAR-T cell improvement. The combination of such powerful technologies is expected to provide a new generation of CAR-T cell-based immunotherapies for clinical application. [ABSTRACT FROM AUTHOR]

Published
2020
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15. Somatic Reversion of a Novel IL2RG Mutation Resulting in Atypical X-Linked Combined Immunodeficiency.

Author

Hou, Yujuan, Gratz, Hans Peter, Ureña-Bailén, Guillermo, Gratz, Paul G., Schilbach-Stückle, Karin, Renno, Tina, Güngör, Derya, Mader, Daniel A., Malenke, Elke, Antony, Justin S., Handgretinger, Rupert, and Mezger, Markus

Subjects
SEVERE combined immunodeficiency, SOMATIC embryogenesis, MISSENSE mutation, IMMUNODEFICIENCY, NUCLEOTIDE sequencing, T cells, GENETIC mutation, INTERLEUKIN-2
Abstract

Mutations of the IL2RG gene, which encodes for the interleukin-2 receptor common gamma chain (γC, CD132), can lead to X-linked severe combined immunodeficiency (X-SCID) associated with a T−B+NK− phenotype as a result of dysfunctional γC-JAK3-STAT5 signaling. Lately, hypomorphic mutations of the IL2RG gene have been described causing atypical SCID with a milder phenotype. Here, we report three brothers with low-normal lymphocyte counts and susceptibility to recurrent respiratory infections and cutaneous warts. The clinical presentation combined with dysgammaglobulinemia suspected an inherited immunity disorder, which has been proven by Next Generation Sequencing as a novel c.458T > C; p.Ile153Thr IL2RG missense-mutation. Subsequent functional characterization revealed impaired T-cell proliferation, low TREC levels and a skewed TCR Vβ repertoire in all three patients. Interestingly, investigation of various subpopulations showed normal expression of CD132 but with partially impaired STAT5 phosphorylation compared to healthy controls. Additionally, we performed precise genetic analysis of subpopulations revealing spontaneous somatic reversion, predominately in lymphoid derived CD3+, CD4+ and CD8+ T cells. Our data demonstrate that the atypical SCID phenotype noticed in these three brothers is due to the combination of hypomorphic IL-2RG function and somatic reversion. [ABSTRACT FROM AUTHOR]

Published
2022
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