Y Genes as Medicine The Development of Gene Therapies for Inborn Errors of Immunity

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA(2022)

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摘要
In all, the field of gene therapy possesses great potential to change the way many monogenic diseases, particularly of the immune system, are treated. As improve-ments in genome modification approaches are developed along with improvements in early diagnosis of disease, medical management, and bone marrow conditioning, it is hoped that there can be even better outcomes for those with rare and once fatal diseases.
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关键词
Gene modification,Viral vector gene therapy,Gene editing,Inborn errors of immunity,Primary immunodeficiency
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