Overview of the current status of gene therapy for primary immune deficiencies (PIDs)

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

卷:146

Review

Kuo, Caroline Y.¹  Kohn, Donald B.^1,2,3,4 

[1] Univ Calif Los Angeles, David Geffen Sch Med, Dept Pediat, Los Angeles, CA 90095 USA

[2] Univ Calif Los Angeles, David Geffen Sch Med, Dept Microbiol Immunol & Mol Genet, Los Angeles, CA 90095 USA

[3] Univ Calif Los Angeles, David Geffen Sch Med, Dept Mol & Med Pharmacol, Los Angeles, CA 90095 USA

[4] Univ Calif Los Angeles, David Geffen Sch Med, Eli & Edythe Broad, Ctr Regenerat Med & Stem Cell Res, Los Angeles, CA 90095 USA

关键词: Primary immune deficiencies;    gene therapy;    lentiviral vectors;    gene editing;    autologous hematopoietic stem cell transplantation;   

DOI  :  10.1016/j.jaci.2020.05.024

来源: Elsevier

【 摘 要 】

Over 3 decades, gene therapy has advanced from a logical idea to becoming a clinical reality for several of the most severe primary immune deficiencies, as well as other inherited disorders. The first gene therapy medicines have been licensed for marketing and several more are advancing toward that goal to make them widely available, beyond clinical trials. Although common platforms of cells, vectors, or editing reagents are used for these disorders, each individual genetic cause of an immune deficiency requires its own vector or editing tools and a package of preclinical data on efficacy and safety to initiate clinical trials. One-by-one, gene therapy for primary immune deficiencies is being brought to the clinic and hopefully will provide safe and effective therapies.

【 授权许可】

Free