Targeted gene modifications in drug discovery and development

被引:10
作者
Cabaniols, Jean-Pierre [1 ]
Mathis, Luc [1 ]
Delenda, Christophe [1 ]
机构
[1] Cellectis BioRes, F-93230 Romainville, France
关键词
THROUGHPUT SCREENING ASSAY; HOMOLOGOUS RECOMBINATION; RNA-INTERFERENCE; MAMMALIAN-CELLS; EXPRESSION; RECEPTOR; BACULOVIRUS; TRANSPORTER; PROTEIN; IDENTIFICATION;
D O I
10.1016/j.coph.2009.05.001
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
In the present review, we discuss technologies and principles guiding the choice of cell-based assays. We show that a major trend is to expedite cell line development in cellular systems [stem cells, induced pluripotent stem cells (iPS), primary cells...] that may reflect the physiology and differentiation of cells in living organs. We discuss different expression technologies and propose that targeted gene integration is the best approach, applicable to all types of cells. We propose that targeted gene expression combined with the development of assays that approach the physiology of the human body, by creating better screening tools, may help to reduce failure and attrition rate at late stages of clinical development.
引用
收藏
页码:657 / 663
页数:7
相关论文
共 65 条
[61]   Detection of potential (anti)progestagenic endocrine disruptors using a recombinant human progesterone receptor binding and transactivation assay [J].
Viswanath, Gunda ;
Halder, Sujata ;
Divya, Gunda ;
Majumder, Chandrajeet B. ;
Roy, Partha .
MOLECULAR AND CELLULAR ENDOCRINOLOGY, 2008, 295 (1-2) :1-9
[62]   An SMA project report: Neural cell-based assays derived from human embryonic stem cells [J].
Wilson, Patricia G. ;
Cherry, Jonathan J. ;
Schwamberger, Shelle ;
Adams, Allison M. ;
Zhou, Jianhua ;
Shin, Soojung ;
Stice, Steven L. .
STEM CELLS AND DEVELOPMENT, 2007, 16 (06) :1027-1041
[63]   Mesenchymal Stem Cells as a Gene Delivery System to Create Biological Pacemaker Cells in vitro [J].
Yang, X-J ;
Zhou, Y. F. ;
Li, H-X ;
Han, L-H ;
Jiang, W-P .
JOURNAL OF INTERNATIONAL MEDICAL RESEARCH, 2008, 36 (05) :1049-1055
[64]   Using multiplexed regulation of luciferase activity and GFP translocation to screen for FOXO modulators [J].
Zanella, Fabian ;
Rosado, Aranzazu ;
Garcia, Beatriz ;
Carnero, Amancio ;
Link, Wolfgang .
BMC CELL BIOLOGY, 2009, 10
[65]   Role of carboxylesterase 1 and impact of natural genetic variants on the hydrolysis of trandolapril [J].
Zhu, Hao-Jie ;
Appel, Dauid I. ;
Johnson, Julie A. ;
Chavin, Kenneth D. ;
Markowitz, John S. .
BIOCHEMICAL PHARMACOLOGY, 2009, 77 (07) :1266-1272