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Keywords: CRISPR/Cas9
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Journal Articles
Dis Model Mech (2024) 17 (6): dmm050524.
Published: 16 April 2024
... in the oocytes. We then mapped a suppressor candidate gene, lmbr-1 , which is an ortholog of human limb development membrane protein 1 ( LMBR1 ). The CRISPR/Cas9 edited lmbr-1 suppressor alleles, lmbr-1(S647F) and lmbr-1(P314L) , both significantly suppressed embryonic lethality and defective eggshell formation...
Includes: Supplementary data
Journal Articles
Journal Articles
Dis Model Mech (2023) 16 (7): dmm050145.
Published: 20 July 2023
... anomalies, diarrhea and thrombocytopenia, and suffered early demise from sepsis. Her consanguineous parents also had a previous child who died with similar clinical features. Using CRISPR/Cas9-mediated approaches, we demonstrate that loss of ARPC5 affects actin cytoskeleton organization and function...
Includes: Supplementary data
Journal Articles
Journal Articles
Dis Model Mech (2020) 13 (10): dmm045096.
Published: 12 October 2020
... response in patient-derived fibroblasts, but not in iPSC-derived cortical neurons. CHIP/STUB1 Cortical neurons CRISPR/Cas9 Heat shock response Induced pluripotent stem cells SCAR16 Mutations in STUB1 (STIP1 homology and U-box containing protein 1) cause early-onset autosomal recessive...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2019) 12 (10): dmm039982.
Published: 16 October 2019
...Yu Kang; Chu Chu; Fang Wang; Yuyu Niu ABSTRACT Owing to their high similarity to humans, non-human primates (NHPs) provide an exceedingly suitable model for the study of human disease. In this Review, we summarize the history of transgenic NHP models and the progress of CRISPR/Cas9-mediated genome...
Journal Articles
In collection:
Cancer
Dis Model Mech (2019) 12 (9): dmm040386.
Published: 6 September 2019
.... the PiggyBac system) for stable random integration of oncogene expression cassettes ( Ding et al., 2005 ). The emergence of CRISPR/Cas9 technology has transformed many areas of biology, including cancer research ( Hsu et al., 2014 ; Wright et al., 2016 ). Genome editing with CRISPR/Cas9 now enables...
Journal Articles
Dis Model Mech (2018) 11 (11): dmm035048.
Published: 20 November 2018
... and function with organoids derived from human pluripotent stem cells. These have successfully recapitulated not only kidney differentiation, but also the specific phenotypical traits related to kidney function. The combination of this methodology with CRISPR/Cas9 genome editing has already helped researchers...
Journal Articles
Dis Model Mech (2018) 11 (10): dmm037515.
Published: 24 October 2018
...Sergey V. Prykhozhij; Jason N. Berman ABSTRACT The zebrafish is an increasingly popular model organism for human genetic disease research. CRISPR/Cas9-based approaches are currently used for multiple gene-editing purposes in zebrafish, but few studies have developed reliable ways to introduce...
Journal Articles
Journal Articles
Journal Articles
Dis Model Mech (2018) 11 (10): dmm036632.
Published: 10 October 2018
... regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 system (CRISPR/Cas9) was used to disrupt the ApoE gene in Bama miniature pigs. Biallelic-modified ApoE pigs with in-frame mutations ( ApoE m/m ) and frameshift mutations ( ApoE −/− ) were simultaneously produced. ApoE −/− pigs...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2018) 11 (10): dmm033852.
Published: 8 October 2018
... atherosclerosis development in the NOD strain is of particular interest as human atherosclerosis on the diabetic and autoimmune background has not been successfully modeled. In this study, we used CRISPR/Cas9 genome editing to genetically disrupt apolipoprotein E (ApoE) and low-density lipoprotein receptor (LDLR...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2018) 11 (6): dmm032631.
Published: 27 June 2018
... due to loss of RNaseT2 function. Diffusion-weighted imaging Magnetic resonance imaging CRISPR/Cas9 Lysosomal storage disease Object recognition Glial fibrillary acidic protein Ribonucleases (RNases) are ubiquitously expressed enzymes which catalyze the cleavage of RNA and function...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2018) 11 (4): dmm031542.
Published: 9 April 2018
...-terminus of fibrillin-1, the penultimate exon of the FBN1 gene. Here, we describe the generation of a rabbit MPL model with C-terminal truncation of fibrillin-1 using a CRISPR/Cas9 system. FBN1 heterozygous ( FBN1 Het) rabbits faithfully recapitulated the phenotypes of MFS, including muscle wasting...
Includes: Supplementary data