Gene therapy comes of age

被引：995

作者：

Dunbar, Cynthia E. ^{[1
]}

High, Katherine A. ^{[2
]}

Joung, J. Keith ^{[3
,4
]}

Kohn, Donald B. ^{[5
]}

Ozawa, Keiya ^{[6
]}

Sadelain, Michel ^{[7
]}

机构：

[1] NHLBI, Hematol Branch, Bethesda, MD 20892 USA

[2] Spark Therapeut, Philadelphia, PA USA

[3] Massachusetts Gen Hosp, Boston, MA 02114 USA

[4] Harvard Med Sch, Boston, MA USA

[5] Univ Calif Los Angeles, David Geffen Sch Med, Los Angeles, CA 90095 USA

[6] Univ Tokyo, Inst Med Sci, Tokyo, Japan

[7] Mem Sloan Kettering Canc Ctr, 1275 York Ave, New York, NY 10021 USA

来源：

SCIENCE | 2018年 / 359卷 / 6372期

关键词：

ADENOASSOCIATED VIRAL VECTORS; CHIMERIC ANTIGEN RECEPTORS; HEMATOPOIETIC STEM-CELLS; HEMOPHILIA-B DOGS; FACTOR-IX; T-CELLS; PARKINSONS-DISEASE; RETROVIRAL VECTORS; IMMUNE-RESPONSES; MOUSE MODEL;

D O I：

10.1126/science.aan4672

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells. We also discuss emerging genome editing technologies that should further advance the scope and efficacy of gene therapy approaches.

引用

页码：175 / +

页数：11

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