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Frontiers in Immunology|September 26, 2022
Partial human Janus kinase 1 deficiency predominantly impairs responses to interferon gamma and intracellular control of mycobacteriaVanessa Daza-Cajigal, Adriana S Albuquerque, Dan F Young, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|August 15, 2006
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioningH Bobby Gaspar, Emma Bjorkegren, Kate Parsley, et al.Nature Communications|November 18, 2017
Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cellsPamela P Lee, Damián Lobato-Márquez, Nayani Pramanik, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 26, 2021
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learningAdrian Schwarzer, Steven R Talbot, Anton Selich, et al.Molecular Therapy. Methods & Clinical Development|August 11, 2020
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe DiseaseGiuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, et al.Molecular Therapy. Methods & Clinical Development|December 12, 2018
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving FunctionalityMarti Cabanes-Creus, Samantha L Ginn, Anais K Amaya, et al.Cell Reports. Medicine|May 14, 2026
Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich's ataxia modelsJeffrey Pido-Lopez, Shefta E Moula, Enas Shaban, et al.Emerging Infectious Diseases|May 29, 2010
Astrovirus encephalitis in boy with X-linked agammaglobulinemiaPhenix Lan Quan, Thor A Wagner, Thomas Briese, et al.Journal of Clinical Immunology|February 23, 2020
Correction to: Human Inborn Errors of Immunity: 2019 Update on the Classification from the International Union of Immunological Societies Expert CommitteeStuart G Tangye, Waleed Al-Herz, Aziz Bousfiha, et al.Journal of Clinical Immunology|January 19, 2020
Human Inborn Errors of Immunity: 2019 Update on the Classification from the International Union of Immunological Societies Expert CommitteeStuart G Tangye, Waleed Al-Herz, Aziz Bousfiha, et al.Pageof 52