1-20 of 21
Keywords: Genome editing
Close
Follow your search
Access your saved searches in your account

Would you like to receive an alert when new items match your search?
Close Modal
Sort by
Journal Articles
Dis Model Mech (2023) 16 (10): dmm050279.
Published: 5 October 2023
... , such as a proliferative advantage and increased organoid budding. Transcriptional profiling revealed differential expression of genes in pathways commonly known to be associated with PTEN loss, including mTORC1 activation. Summary: High-efficiency ribonucleoprotein-mediated genome editing enables rapid functional...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2022) 15 (6): dmm049463.
Published: 6 July 2022
... a genome-editing strategy to induce red blood cell fetal hemoglobin (HbF; α 2 γ 2 ). Berkeley SCD mice contain four to 22 randomly arranged, fragmented copies of three human transgenes ( HBA1 , HBG2-HBG1-HBD-HBB S and a mini-locus control region) integrated into a single site of mouse chromosome 1. Cas9...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2022) 15 (6): dmm049463.
Published: 6 July 2022
... a genome-editing strategy to induce red blood cell fetal hemoglobin (HbF; α 2 γ 2 ). Berkeley SCD mice contain four to 22 randomly arranged, fragmented copies of three human transgenes ( HBA1 , HBG2-HBG1-HBD-HBB S and a mini-locus control region) integrated into a single site of mouse chromosome 1. Cas9...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2019) 12 (10): dmm039982.
Published: 16 October 2019
... shortcomings of the application of the CRISPR/Cas9 system to NHPs that have emerged from the expanded understanding of this technology in recent years. Although off-target and mosaic mutations are the main concerns in CRISPR/Cas9-mediated NHP modeling, recent progress in genome editing techniques make...
Journal Articles
Dis Model Mech (2019) 12 (10): dmm039982.
Published: 16 October 2019
... shortcomings of the application of the CRISPR/Cas9 system to NHPs that have emerged from the expanded understanding of this technology in recent years. Although off-target and mosaic mutations are the main concerns in CRISPR/Cas9-mediated NHP modeling, recent progress in genome editing techniques make...
Journal Articles
Dis Model Mech (2020) 13 (2): dmm041673.
Published: 1 October 2019
... testing of the on-target efficiency of human-specific CRISPR/Cas9 systems, they do not allow accurate assessment of off-target effects, as only the transgene has been humanized. Humanized animal models for the evaluation of splice modulating or genome editing therapies need to fulfill several...
Journal Articles
Dis Model Mech (2020) 13 (2): dmm041673.
Published: 1 October 2019
... attributed. Summary: Personalized medicine approaches benefit from humanized animal models. Here, we outline the usefulness, caveats and considerations for generating and using these models for pre-clinical studies of Duchenne muscular dystrophy. Genetic therapies Genome editing Exon skipping...
Journal Articles
Dis Model Mech (2019) 12 (5): dmm035790.
Published: 7 May 2019
... ) gene are common genetic abnormalities in human myeloid malignancies and induce clonal expansion of mutated hematopoietic stem cells (HSCs). To understand how ASXL1 disruption leads to myeloid cell transformation, we generated asxl1 haploinsufficient and null zebrafish lines using genome-editing...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2019) 12 (5): dmm035790.
Published: 7 May 2019
... ) gene are common genetic abnormalities in human myeloid malignancies and induce clonal expansion of mutated hematopoietic stem cells (HSCs). To understand how ASXL1 disruption leads to myeloid cell transformation, we generated asxl1 haploinsufficient and null zebrafish lines using genome-editing...
Includes: Supplementary data
Journal Articles
Journal Articles
Journal Articles
Dis Model Mech (2019) 12 (3): dmm039370.
Published: 7 March 2019
... rates of DNA repair ( Boel et al., 2018 ). Capturing the excitement of gene editing in zebrafish, an editorial by the Berman laboratory provides an up-to-date view of the state of the art for genome editing in zebrafish ( Prykhozhij and Berman, 2018 ). The broad spectrum of subjects...
Journal Articles
Dis Model Mech (2019) 12 (2): dmm039271.
Published: 22 February 2019
.... Rare disease Disease model Face validity Genome editing Clinical translation Disease Models and Mechanisms (DMM) is a journal dedicated to improving human health through publishing research in model systems. To this end, DMM publishes articles that develop and validate models...
Journal Articles
Dis Model Mech (2019) 12 (1): dmm029462.
Published: 8 January 2019
... ). Dll4–Notch signaling in Flt3-independent dendritic cell development and autoimmunity in mice . J. Exp. Med.   209 , 1011 - 1028 . 10.1084/jem.20111615 Birling , M.-C. , Herault , Y. and Pavlovic , G. ( 2017 ). Modeling human disease in rodents by CRISPR/Cas9 genome editing...
Includes: Supplementary data
Journal Articles
Journal Articles
Journal Articles
Journal Articles
Dis Model Mech (2018) 11 (10): dmm037515.
Published: 24 October 2018
.... and Schmid , B. ( 2013 ). Efficient CRISPR/Cas9 genome editing with low off-target effects in zebrafish . Development   140 , 4982 - 4987 . 10.1242/dev.099085 Hwang , W. Y. , Fu , Y. , Reyon , D. , Maeder , M. L. , Kaini , P. , Sander , J. D. , Joung , J. K...
Journal Articles
Dis Model Mech (2018) 11 (10): dmm037515.
Published: 24 October 2018
...-based approaches for knocking in mutations in zebrafish ( dmm035352 , dmm035469 and dmm035972 ), this Editorial reflects on the current state of genome editing for disease modeling in zebrafish. CRISPR/Cas9 Genome editing Point mutations Zebrafish One of the great potentials...
Journal Articles