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Current status and prospects of Primary immunodeficiency diseases in Asia

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Genes & diseases 2020年第1期7卷 3-11页

作者： Rakesh Kumar Pilania Himanshi Chaudhary Ankur Kumar Jindal Amit Rawat Surjit Singh Allergy Immunology Unit Department of PediatricsAdvanced Pediatrics CentrePostgraduate Institute of Medical Education and ResearchChandigarh160012India

Primary immunodeficiency diseases(PIDs)are increasingly being reported across the World.Several advances have been made in the diagnostic and therapeutic research related to PIDs.With increasing awareness,the field of PIDs has rapidly evolved in Asia as well.In this review,we summarize the progress that has been made in the field of PIDs in Asian countries;major limitations and challenges faced by the clinicians working in this field in Asia;difference in spectrum of PIDs in Asia from rest of the World;current state of diagnostic and treatment facilities available in various countries in Asia and the future prospects of these diseases in the continent.

关键词： Asia Genetic diagnosis Hematopoietic stem cell transplant Intravenous immunoglobulin Primary immunodeficiency diseases

An updated review on phenocopies of Primary immunodeficiency diseases

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Genes & diseases 2020年第1期7卷 12-25页

作者： Ankita Singh Ankur K.Jindal Vibhu Joshi Gummadi Anjani Amit Rawat Allergy Immunology Unit Department of PediatricsAdvanced Pediatrics CentrePostgraduate Institute of Medical Education and ResearchChandigarhIndia

Primary immunodeficiency diseases(PIDs)refer to a heterogenous group of disorders characterized clinically by increased susceptibility to infections,autoimmunity and increased risk of malignancies.These group of disorders present with clinical manifestations similar to PIDs with known genetic defects but have either no genetic defect or have a somatic mutation and thus have been labelled as“Phenocopies of PIDs”.These diseases have been further subdivided into those associated with somatic mutations and those associated with presence of auto-antibodies against various cytokines.In this review,we provide an update on clinical manifestations,diagnosis and management of these diseases.

关键词： Anti-cytokine antibodies Phenocopies Primary immunodeficiency diseases Somatic mutations

Recent advances in lentiviral vectors for gene therapy

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Science China(Life Sciences) 2021年第11期64卷 1842-1857页

作者： Xiaoyu Wang Cuicui Ma Roberto Rodríguez Labrada Zhou Qin Ting Xu Zhiyao He Yuquan Wei Department of Pharmacy State Key Laboratory of Biotherapy and Cancer CenterNational Clinical Research Center for GeriatricsWest China HospitalSichuan UniversityChengdu 610041China Department Clinical Neurophysiology Centre for the Research and Rehabilitation of Hereditary AtaxiasHolguín 80100Cuba Key Laboratory of Drug-Targeting and Drug Delivery System of the Education Ministry Sichuan Engineering Laboratory for Plant-Sourced Drug and Sichuan Research Center for Drug Precision Industrial TechnologyWest China School of PharmacySichuan UniversityChengdu 610041China

Lentiviral vectors(LVs), derived from human immunodeficiency virus, are powerful tools for modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells. With the extensive and in-depth studies on this gene therapy vehicle over the past two decades, LVs have been widely used in both research and clinical trials. For instance, third-generation and selfinactive LVs have been used to introduce a gene with therapeutic potential into the host genome and achieve targeted delivery into specific tissue. When LVs are employed in leukemia, the transduced T cells recognize and kill the tumor B cells;in β-thalassemia, the transduced CD34^(+) cells express normal β-globin;in adenosine deaminase-deficient severe combined immunodeficiency, the autologous CD34^(+) cells express adenosine deaminase and realize immune reconstitution. Overall, LVs can perform significant roles in the treatment of Primary immunodeficiency diseases, hemoglobinopathies, B cell leukemia, and neurodegenerative diseases. In this review, we discuss the recent developments and therapeutic applications of LVs. The safe and efficient LVs show great promise as a tool for human gene therapy.

关键词： lentiviral vector gene therapy Primary immunodeficiency diseases leukemia hemoglobinopathies neurodegenerative diseases

Druggable monogenic immune defects hidden in diverse medical specialties:Focus on overlap syndromes

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World Journal of Clinical Pediatrics 2022年第2期11卷 136-150页

作者： Valentina Boz Chiara Zanchi Laura Levantino Guglielmo Riccio Alberto Tommasini Department of Medical Surgical and Health SciencesUniversity of TriesteTrieste 34137Italy Department of Pediatrics Institute for Maternal and Child HealthIRCCS Burlo GarofoloTrieste 34137Italy

In the last two decades two new paradigms changed our way of perceiving primary immunodeficiencies:An increasing number of immune defects are more associated with inflammatory or autoimmune features rather than with infections.Some primary immune defects are due to hyperactive pathways that can be targeted by specific inhibitors,providing innovative precision treatments that can change the natural history of diseases.In this article we review some of these“druggable”inborn errors of immunity and describe how they can be suspected and diagnosed in diverse pediatric and adult medicine specialties.Since the availability of precision treatments can dramatically impact the course of these diseases,preventing the development of organ damage,it is crucial to widen the awareness of these conditions and to provide practical hints for a prompt detection and cure.

关键词： Inborn errors of immunity Primary immunodeficiency diseases Precision treatments Immunodysregulation Autoimmunity Overlap syndromes