Use of Catalytic DNA in Target Validation and Therapeutics

2004 ◽  
pp. 65-79
Author(s):  
Lun-Quan Sun
Keyword(s):  
Target Validation ◽  
Catalytic Dna

Pathology and Development - Developmental Systems for Target Validation and Drug Screening in Osteoarthritis

2005 ◽  
Vol 2 (6) ◽  
pp. 435-448 ◽  
Author(s):  
N. Gerwin ◽  
T. Pellas ◽  
U. Junker ◽  
T. Aigner
Keyword(s):  
Drug Screening ◽  
Target Validation ◽  
Developmental Systems

scholarly journals Rapid target validation in a Cas9-inducible hiPSC derived kidney model

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Yasaman Shamshirgaran ◽  
Anna Jonebring ◽  
Anna Svensson ◽  
Isabelle Leefa ◽  
Mohammad Bohlooly-Y ◽  
...  
Keyword(s):  
High Efficiency ◽  
Disease Modeling ◽  
Genetic Manipulation ◽  
Disease Model ◽  
Genetically Engineered ◽  
Model Systems ◽  
Target Validation ◽  
Direct Differentiation ◽  
Kidney Organoids

AbstractRecent advances in induced pluripotent stem cells (iPSCs), genome editing technologies and 3D organoid model systems highlight opportunities to develop new in vitro human disease models to serve drug discovery programs. An ideal disease model would accurately recapitulate the relevant disease phenotype and provide a scalable platform for drug and genetic screening studies. Kidney organoids offer a high cellular complexity that may provide greater insights than conventional single-cell type cell culture models. However, genetic manipulation of the kidney organoids requires prior generation of genetically modified clonal lines, which is a time and labor consuming procedure. Here, we present a methodology for direct differentiation of the CRISPR-targeted cell pools, using a doxycycline-inducible Cas9 expressing hiPSC line for high efficiency editing to eliminate the laborious clonal line generation steps. We demonstrate the versatile use of genetically engineered kidney organoids by targeting the autosomal dominant polycystic kidney disease (ADPKD) genes: PKD1 and PKD2. Direct differentiation of the respective knockout pool populations into kidney organoids resulted in the formation of cyst-like structures in the tubular compartment. Our findings demonstrated that we can achieve > 80% editing efficiency in the iPSC pool population which resulted in a reliable 3D organoid model of ADPKD. The described methodology may provide a platform for rapid target validation in the context of disease modeling.

Screening the receptorome: an efficient approach for drug discovery and target validation

2006 ◽  
Vol 11 (15-16) ◽  
pp. 708-716 ◽  
Author(s):  
Ryan T. Strachan ◽  
Gina Ferrara ◽  
Bryan L. Roth
Keyword(s):  
Drug Discovery ◽  
Target Validation ◽  
Efficient Approach

High-throughput target validation in model organisms

2004 ◽  
Vol 3 (5) ◽  
pp. 191-197 ◽  
Author(s):  
Thanh N. Doan ◽  
Carmen D. Eilertson ◽  
Amy L. Rubinstein
Keyword(s):  
High Throughput ◽  
Model Organisms ◽  
Target Validation

scholarly journals Examination of Bacterial Inhibition Using a Catalytic DNA

PLoS ONE ◽  
2014 ◽  
Vol 9 (12) ◽  
pp. e115640 ◽  
Author(s):  
Long Qu ◽  
M. Monsur Ali ◽  
Sergio D. Aguirre ◽  
Hongxia Liu ◽  
Yuyang Jiang ◽  
...  
Keyword(s):  
Bacterial Inhibition ◽  
Catalytic Dna
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