Gene therapy comes of age

被引：1037

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

共 155 条

[71] CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes [J].

Komor, Alexis C. ;

Badran, Ahmed H. ;

Liu, David R. .

CELL, 2017, 168 (1-2) :20-36

[72] Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage [J].

Komor, Alexis C. ;

Kim, Yongjoo B. ;

Packer, Michael S. ;

Zuris, John A. ;

Liu, David R. .

NATURE, 2016, 533 (7603) :420-+

[73] Progression to the G1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T cells [J].

Korin, YD ;

Zack, JA .

JOURNAL OF VIROLOGY, 1998, 72 (04) :3161-3168

[74] Viral Vectors for Gene Therapy: Translational and Clinical Outlook [J].

Kotterman, Melissa A. ;

Chalberg, Thomas W. ;

Schaffer, David V. .

ANNUAL REVIEW OF BIOMEDICAL ENGINEERING, VOL 17, 2015, 17 :63-89

[75] T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial [J].

Lee, Daniel W. ;

Kochenderfer, James N. ;

Stetler-Stevenson, Maryalice ;

Cui, Yongzhi K. ;

Delbrook, Cindy ;

Feldman, Steven A. ;

Fry, Terry J. ;

Orentas, Rimas ;

Sabatino, Marianna ;

Shah, Nirali N. ;

Steinberg, Seth M. ;

Stroncek, Dave ;

Tschemia, Nick ;

Yuan, Constance ;

Zhang, Hua ;

Zhang, Ling ;

Rosenberg, Steven A. ;

Wayne, Alan S. ;

Mackall, Crystal L. .

LANCET, 2015, 385 (9967) :517-528

[76] Current concepts in the diagnosis and management of cytokine release syndrome [J].

Lee, Daniel W. ;

Gardner, Rebecca ;

Porter, David L. ;

Louis, Chrystal U. ;

Ahmed, Nabil ;

Jensen, Michael ;

Grupp, Stephan A. ;

Mackall, Crystal L. .

BLOOD, 2014, 124 (02) :188-195

[77] AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial [J].

LeWitt, Peter A. ;

Rezai, Ali R. ;

Leehey, Maureen A. ;

Ojemann, Steven G. ;

Flaherty, Alice W. ;

Eskandar, Emad N. ;

Kostyk, Sandra K. ;

Thomas, Karen ;

Sarkar, Atom ;

Siddiqui, Mustafa S. ;

Tatter, Stephen B. ;

Schwalb, Jason M. ;

Poston, Kathleen L. ;

Henderson, Jaimie M. ;

Kurlan, Roger M. ;

Richard, Irene H. ;

Van Meter, Lori ;

Sapan, Christine V. ;

During, Matthew J. ;

Kaplitt, Michael G. ;

Feigin, Andrew .

LANCET NEUROLOGY, 2011, 10 (04) :309-319

[78] Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger Nucleases [J].

Li, Lijing ;

Krymskaya, Ludmila ;

Wang, Jianbin ;

Henley, Jill ;

Rao, Anitha ;

Cao, Lan-Feng ;

Chy-Anh Tran ;

Torres-Coronado, Monica ;

Gardner, Agnes ;

Gonzalez, Nancy ;

Kim, Kenneth ;

Liu, Pei-Qi ;

Hofer, Ursula ;

Lopez, Evan ;

Gregory, Philip D. ;

Liu, Qing ;

Holmes, Michael C. ;

Cannon, Paula M. ;

Zaia, John A. ;

DiGiusto, David L. .

MOLECULAR THERAPY, 2013, 21 (06) :1259-1269

[79] TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain [J].

Li, Ting ;

Huang, Sheng ;

Jiang, Wen Zhi ;

Wright, David ;

Spalding, Martin H. ;

Weeks, Donald P. ;

Yang, Bing .

NUCLEIC ACIDS RESEARCH, 2011, 39 (01) :359-372

[80] CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes [J].

Liang, Puping ;

Xu, Yanwen ;

Zhang, Xiya ;

Ding, Chenhui ;

Huang, Rui ;

Zhang, Zhen ;

Lv, Jie ;

Xie, Xiaowei ;

Chen, Yuxi ;

Li, Yujing ;

Sun, Ying ;

Bai, Yaofu ;

Songyang, Zhou ;

Ma, Wenbin ;

Zhou, Canquan ;

Huang, Junjiu .

PROTEIN & CELL, 2015, 6 (05) :363-372

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