Gene Therapy for Primary Immunodeficiencies

作者：Fischer Alain^*; Hacein Bey Abina S; Cavazzana Calvo M

来源：Hematology-Oncology Clinics of North America, 2011, 25(1): 89-+.

DOI：10.1016/j.hoc.2010.11.007

• Severe combined immunodeficiencies
• Gene therapy
• Hematopoietic stem cells
• Retrovirus
• Lentivirus
• Wiskott Aldrich
• Chronic granulomatous disease
• Clinical trial

The concept of gene therapy emerged as a way of correcting monogenic inherited diseases by introducing a normal copy of the mutated gene into at least some of the patients' cells. Although this concept has turned out to be quite complicated to implement, it is in the field of primary immunodeficiencies (PIDs) that proof of feasibility has been undoubtedly achieved. There is now a strong rationale in support of gene therapy for at least some PIDs, as discussed in this article.

• 出版日期2011-2