Gene therapy for inborn errors of immunity: Base editing comes into play

被引:3
|
作者
Malech, Harry L. [1 ]
Notarangelo, Luigi D. [1 ]
机构
[1] NIH, Natl Inst Allergy & Infect Dis, Lab Clin Immunol & Microbiol, Bethesda, MD 20892 USA
基金
美国国家卫生研究院;
关键词
GENERATION;
D O I
10.1016/j.cell.2023.03.001
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
CRISPR-Cas9-based base editing allows precise base editing to achieve conversion of adenosine to guanine or cytosine to thymidine. In this issue of Cell, McAuley et al. use adenine base editing to correct a single base-pair mutation causing human CD3d deficiency, demonstrating superior efficiency of genetic correction with reduced undesired genetic alterations compared with standard CRISPR-Cas9 editing.
引用
收藏
页码:1302 / 1304
页数:3
相关论文
共 50 条
  • [31] Editorial: Inborn errors of immunity and mucosal immunity
    Hsieh, Elena Wen-Yuan
    Snapper, Scott B.
    de Zoeten, Edwin F.
    FRONTIERS IN IMMUNOLOGY, 2023, 14
  • [32] Liver immunity, autoimmunity, and inborn errors of immunity
    Yavuz Emre Parlar
    Sefika Nur Ayar
    Deniz Cagdas
    Yasemin H Balaban
    World Journal of Hepatology, 2023, 15 (01) : 52 - 67
  • [33] Liver immunity, autoimmunity, and inborn errors of immunity
    Parlar, Yavuz Emre
    Ayar, Sefika Nur
    Cagdas, Deniz
    Balaban, Yasemin H.
    WORLD JOURNAL OF HEPATOLOGY, 2023, 15 (01) : 52 - 67
  • [34] We play "Inborn Errors of Metabolism"
    Mkheidze, M
    EUROPEAN JOURNAL OF HUMAN GENETICS, 1998, 6 : 179 - 179
  • [35] Y Genes as Medicine The Development of Gene Therapies for Inborn Errors of Immunity
    Long, Joseph D.
    Trope, Edward C.
    Yang, Jennifer
    Rector, Kristen
    Kuo, Caroline Y.
    HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA, 2022, 36 (04) : 829 - 851
  • [36] Score-Based Candidate Gene Identification in Inborn Errors of Immunity
    Dudler, Claire
    Mueller-Nedebock, Amica
    Fasshauer, Maria
    Borte, Stephan
    Gerisch, Marie
    Abou Jamra, Rami
    Schumann, Isabell
    EUROPEAN JOURNAL OF HUMAN GENETICS, 2024, 32 : 1425 - 1425
  • [37] Improving Access to Therapy for Patients With Inborn Errors of Immunity: A Call to Action
    Dorsey, Morna J.
    Condino-Neto, Antonio
    JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE, 2023, 11 (06): : 1698 - 1702
  • [38] Repercussions of inborn errors of immunity on growth
    Goudouris, Ekaterini Simioes
    Silva Segundo, Gesmar Rodrigues
    Poli, Cecilia
    JORNAL DE PEDIATRIA, 2019, 95 : S49 - S58
  • [39] Viral infections and inborn errors of immunity
    Ewing, Anne
    Madan, Rebecca Pellett
    CURRENT OPINION IN INFECTIOUS DISEASES, 2024, 37 (04) : 227 - 231
  • [40] Inborn Errors of Immunity and Coronovirus Pandemic
    Camcioglu, Yildiz
    TURKISH JOURNAL OF IMMUNOLOGY, 2020, 8 (01): : 13 - 20