Gene therapy strategies for RAG1 deficiency: Challenges and breakthroughs.

Autor: Gilioli G; Department of Immunology, Leiden University Medical Center, Leiden, the Netherlands., Lankester AC; Department of Pediatrics, Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, the Netherlands., de Kivit S; Department of Immunology, Leiden University Medical Center, Leiden, the Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden, the Netherlands. Electronic address: F.J.T.Staal@lumc.nl., Ott de Bruin LM; Department of Immunology, Leiden University Medical Center, Leiden, the Netherlands; Department of Pediatrics, Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, the Netherlands.

Publikováno v: Immunology letters [Immunol Lett] 2024 Dec; Vol. 270, pp. 106931. Date of Electronic Publication: 2024 Sep 18.

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.

Autor: Castiello MC; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Di Verniere M; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Draghici E; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Fontana E; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Clinical and Research Center, IRCCS, Rozzano, Milan, Italy., Penna S; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Sereni L; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Zecchillo A; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Minuta D; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Uva P; Clinical Bioinformatics, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Zahn M; ProtaGene CGT GmbH, Heidelberg, Germany., Gil-Farina I; ProtaGene CGT GmbH, Heidelberg, Germany., Annoni A; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Iaia S; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Ott de Bruin LM; Willem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, Netherlands.; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States., Pike-Overzet K; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands., Villa A; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Capo V; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.

Publikováno v: Frontiers in immunology [Front Immunol] 2023 Nov 13; Vol. 14, pp. 1268620. Date of Electronic Publication: 2023 Nov 13 (Print Publication: 2023).

Cysteine and hydrophobic residues in CDR3 serve as distinct T-cell self-reactivity indices.

Autor: Daley SR; Infection and Immunity Program, Monash Biomedicine Discovery Institute and Department of Biochemistry and Molecular Biology, Monash University, Melbourne, Australia. Electronic address: stephen.daley@monash.edu., Koay HF; Department of Microbiology and Immunology, Peter Doherty Institute for Infection and Immunity, University of Melbourne, Melbourne, Australia; Australian Research Council Centre of Excellence in Advanced Molecular Imaging, University of Melbourne, Melbourne, Australia., Dobbs K; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Bosticardo M; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Wirasinha RC; Infection and Immunity Program, Monash Biomedicine Discovery Institute and Department of Biochemistry and Molecular Biology, Monash University, Melbourne, Australia., Pala F; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Castagnoli R; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Rowe JH; Division of Hematology-Oncology, Boston Children's Hospital and Dana Farber Cancer Institute, Harvard Medical School, Boston, Mass., Ott de Bruin LM; Division of Immunology, Boston Children's Hospital, Harvard Medical School, Boston, Mass., Keles S; Division of Pediatric Immunology and Allergy, Meram Medical Faculty, Necmettin Erbakan University, Konya, Turkey., Lee YN; Immunology Service, Edmond and Lily Safra Children's Hospital Sheba Medical Center, Tel Hashomer, Israel., Somech R; Immunology Service, Edmond and Lily Safra Children's Hospital Sheba Medical Center, Tel Hashomer, Israel., Holland SM; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Delmonte OM; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Draper D; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Maxwell S; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Niemela J; Immunology Service, Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md., Stoddard J; Immunology Service, Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md., Rosenzweig SD; Immunology Service, Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md., Poliani PL; Department of Molecular and Translational Medicine, Pathology Unit, University of Brescia, Brescia, Italy., Capo V; San Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific Institute, Milan, Italy., Villa A; San Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific Institute, Milan, Italy; Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Milan Unit, Milan, Italy., Godfrey DI; Department of Microbiology and Immunology, Peter Doherty Institute for Infection and Immunity, University of Melbourne, Melbourne, Australia; Australian Research Council Centre of Excellence in Advanced Molecular Imaging, University of Melbourne, Melbourne, Australia., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md. Electronic address: luigi.notarangelo2@nih.gov.

Publikováno v: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2019 Jul; Vol. 144 (1), pp. 333-336. Date of Electronic Publication: 2019 Apr 30.

Corrigendum: Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56 ^bright NKG2A ⁺⁺⁺ Cells, and Yet Display Increased Degranulation and Higher Perforin Content.

Autor: Dobbs K; Laboratory of Host Defenses, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States., Tabellini G; Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy., Calzoni E; 'A. Nocivelli Institute for Molecular Medicine', Pediatric Clinic, University of Brescia, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia, Brescia, Italy., Patrizi O; Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy., Martinez P; Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina., Giliani SC; 'A. Nocivelli Institute for Molecular Medicine', Pediatric Clinic, University of Brescia, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia, Brescia, Italy., Moratto D; 'A. Nocivelli Institute for Molecular Medicine', Pediatric Clinic, University of Brescia, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia, Brescia, Italy., Al-Herz W; Department of Pediatrics, Faculty of Medicine, Kuwait University, Kuwait City, Kuwait., Cancrini C; DPUO, Division of Immuno-Infectivology, University Department of Pediatrics, Bambino Gesù Children's Hospital, Rome, Italy.; School of Medicine, University of Tor Vergata, Rome, Italy., Cowan M; Pediatric Allergy Immunology and Blood and Marrow Transplant Division, University of California, San Francisco, Benioff Children's Hospital, San Francisco, CA, United States., Bleesing J; Division of Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States., Booth C; Institute for Immunity and Transplantation, University College London, London, United Kingdom., Buchbinder D; Division of Pediatric Hematology, Children's Hospital Orange County, University of California Irvine, Orange County, CA, United States., Burns SO; Institute for Immunity and Transplantation, University College London, London, United Kingdom.; Department of Immunology, Royal Free London NHS Foundation Trust, London, United Kingdom., Chatila TA; Division of Immunology, Boston Children's Hospital, Boston, MA, United States., Chou J; Division of Immunology, Boston Children's Hospital, Boston, MA, United States., Daza-Cajigal V; Institute for Immunity and Transplantation, University College London, London, United Kingdom., Ott de Bruin LM; Division of Immunology, Boston Children's Hospital, Boston, MA, United States., de la Morena MT; Division of Allergy and Immunology, Southwestern Medical Center, University of Texas, Dallas, TX, United States., Di Matteo G; DPUO, Division of Immuno-Infectivology, University Department of Pediatrics, Bambino Gesù Children's Hospital, Rome, Italy.; School of Medicine, University of Tor Vergata, Rome, Italy., Finocchi A; DPUO, Division of Immuno-Infectivology, University Department of Pediatrics, Bambino Gesù Children's Hospital, Rome, Italy.; School of Medicine, University of Tor Vergata, Rome, Italy., Geha R; Division of Immunology, Boston Children's Hospital, Boston, MA, United States., Goyal RK; Division of Hematology/Oncology/BMT, Children's Mercy Hospital & Clinics, Kansas City, MO, United States., Hayward A; Department of Pediatrics, Brown University, Providence, RI, United States., Holland S; Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States., Huang CH; Department of Paediatrics, National University Hospital, Singapore, Singapore., Kanariou MG; Department of Immunology-Histocompatibility, 'Aghia Sophia' Children's Hospital, Athens, Greece., King A; Division of Pediatric Immunology, Hospital Luis Calvo Mackenna, Santiago, Chile., Kaplan B; Department of Pediatrics, Division of Allergy and Immunology, Hofstra Northwell School of Medicine, Hofstra University, Great Neck, NY, United States., Kleva A; Department of Pediatrics, Division of Allergy and Immunology, Hofstra Northwell School of Medicine, Hofstra University, Great Neck, NY, United States., Kuijpers TW; Department of Pediatric Hematology, Immunology and Infectious Diseases, Emma Children's Hospital, Academic Medical Center (AMC), University of Amsterdam, Amsterdam, Netherlands., Lee BW; Department of Paediatrics, National University Hospital, Singapore, Singapore., Lougaris V; Department of Experimental and Clinical Sciences, University of Brescia, Brescia, Italy., Massaad M; Division of Immunology, Boston Children's Hospital, Boston, MA, United States., Meyts I; Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium., Morsheimer M; Transplantation Branch, Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Rockville, MD, United States., Neven B; Pediatric Hematology-Immunology Department, Hospital Necker-Enfants Malades, Institute Imagine, AP-HP, Paris Descartes University, Sorbonne-Paris-Cité, Paris, France., Pai SY; Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA, United States., Parvaneh N; Division of Allergy and Clinical Immunology, Department of Pediatrics, Tehran University of Medical Sciences, Tehran, Iran.; Research Center for Immunodeficiencies, Children's Medical Center, Tehran, Iran., Plebani A; Department of Experimental and Clinical Sciences, University of Brescia, Brescia, Italy., Prockop S; Bone Marrow Transplant Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, United States., Reisli I; Division of Pediatric Immunology and Allergy, Meram Medical Faculty, Necmettin Erbakan University, Konya, Turkey., Soh JY; Department of Paediatrics, National University Hospital, Singapore, Singapore., Somech R; Pediatric Immunology Unit, The Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel., Torgerson TR; Department of Pediatrics and Immunology, Seattle Children's Hospital, University of Washington, Seattle, WA, United States., Kim YJ; Division of Infectious Diseases and Immunodeficiency, Department of Pediatrics, Samsung Medical Center, School of Medicine, Sungkyunkwan University, Seoul, South Korea., Walter JE; Division of Pediatric Allergy/Immunology, University of South Florida at Johns Hopkins All Children's Hospital, St. Petersburg, FL, United States., Gennery AR; Department of Paediatric Immunology, Great North Children's Hospital, Newcastle Upon Tyne, United Kingdom.; Institute of Cellular Medicine, Newcastle University, Newcastle Upon Tyne, United Kingdom., Keles S; Division of Pediatric Immunology and Allergy, Meram Medical Faculty, Necmettin Erbakan University, Konya, Turkey., Manis JP; Department of Laboratory Medicine, Boston Children's Hospital, Boston, MA, United States., Marcenaro E; Molecular Immunology Laboratories, Department of Experimental Medicine, University of Genoa, Genoa, Italy., Moretta A; Molecular Immunology Laboratories, Department of Experimental Medicine, University of Genoa, Genoa, Italy., Parolini S; Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy., Notarangelo LD; Laboratory of Host Defenses, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.

Publikováno v: Frontiers in immunology [Front Immunol] 2017 Oct 10; Vol. 8, pp. 1244. Date of Electronic Publication: 2017 Oct 10 (Print Publication: 2017).

Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies.

Autor: Ott de Bruin LM; Division of Immunology, Boston Children's Hospital, Harvard Medical School , Boston, MA , USA ; Department of Pediatric Immunology, Wilhelmina Children's Hospital, University Medical Center Utrecht , Utrecht , Netherlands., Volpi S; UO Pediatria 2, Istituto Giannina Gaslini, University of Genoa , Genoa , Italy ; Division of Immunology and Allergy, Laboratory Center of Epalinges (CLE), University Hospital of Lausanne , Epalinges , Switzerland., Musunuru K; Department of Stem Cell and Regenerative Biology, Harvard University , Cambridge, MA , USA.

Publikováno v: Frontiers in immunology [Front Immunol] 2015 May 21; Vol. 6, pp. 250. Date of Electronic Publication: 2015 May 21 (Print Publication: 2015).