A doxycycline-inducible system for genetic correction of iPSC disease models

Methods in Molecular Biology

Volumn 1353, Issue , 2014, Pages 13-23

  Sim, Xiuli ^a   Cardenas Diaz, Fabian L ^a   French, Deborah L ^a   Gadue, Paul ^a  

^a NONE

Author keywords

Disease modeling; Doxycyclineinducible expression; Gene targeting; Genetic correction; Homologous recombination; Zinc finger nucleases

Indexed keywords

CRISPR ASSOCIATED PROTEIN; DOXYCYCLINE; PARVOVIRUS VECTOR; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; ZINC FINGER NUCLEASE; ENDONUCLEASE; GREEN FLUORESCENT PROTEIN; ZINC FINGER PROTEIN;

ALLELE; CELL DIFFERENTIATION; CLONE; DISEASE MODEL; DNA INTEGRATION; DNA STRUCTURE; GENE EXPRESSION SYSTEM; GENE LOCUS; GENE TARGETING; GENE VECTOR; GENETIC MANIPULATION; GENETIC TRANSFECTION; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; PLURIPOTENT STEM CELL; POLYMERASE CHAIN REACTION; PROMOTER REGION; SOUTHERN BLOTTING; TET RESPONSE ELEMENT; ANIMAL; BIOLOGICAL MODEL; CELL LINE; CHEMISTRY; CRISPR CAS SYSTEM; CYTOLOGY; DRUG EFFECTS; FIBROBLAST; GENETICS; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MOUSE; PATHOLOGY; PROCEDURES; REPORTER GENE; TRANSGENE;

ANIMALS; CELL LINE; CRISPR-CAS SYSTEMS; DOXYCYCLINE; ENDONUCLEASES; FIBROBLASTS; GENE TARGETING; GENES, REPORTER; GENETIC LOCI; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HOMOLOGOUS RECOMBINATION; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MICE; MODELS, BIOLOGICAL; PROMOTER REGIONS, GENETIC; TRANSGENES; ZINC FINGERS;

EID: 84946727639     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/7651_2014_179     Document Type: Chapter

References (12)

1. DeKelver RC, Choi VM, Moehle E et al (2010) Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res 20:1133-1142
2. Hockemeyer D, Soldner F, Beard C et al (2009) Efficient targeting ofexpressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27:851-857
3. Lombardo A, Cesana D, Genovese P et al (2011) Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods 8:861-869
4. Smith JR Maguire S, Davis L et al (2008) Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration. Stem cells 26:496-504
5. Tiyaboonchai A, Mac H, Shamsedeen R et al (2014) Utilization of the AAVS1 safe harbor locus for hematopoietic specific transgene expression and gene knockdown in human ES cells. Stem Cell Res 12:630-637
6. Sullivan SK, Mills JA, Koukouritaki SB et al (2014) High-level transgene expression in induced pluripotent stem cell-derived megakaryocytes: correction of Glanzmann thrombasthenia. Blood 123:753-757
7. Carroll D (2011) Genome engineering with zinc-finger nucleases. Genetics 188: 773-782
8. Gaj T, Gersbach CA, Barbas CF III (2013) ZFN, TALEN and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol 31:397-405
9. Qian K, Huang C, Chen H et al (2014) A simple and efficient system for regulating gene expression in human pluripotent stem cells and derivatives. Stem Cells 32:1230-1238
10. Wu Y, Liang D, Wang Y et al (2013) Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell Stem Cell 13:659-662
11. Cho SW, Kim S, Kim JM et al (2013) Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol 31:230-232
12. Mali P, Yang L, Esvelt KM et al (2013) RNA-guided human genome engineering via Cas9. Science 339:823-826