CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression

CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression

Gene and cellular therapies hold tremendous promise as agents for treating genetic disorders. However, the effective delivery of genes, particularly large ones, and expression at therapeutic levels can be challenging in cells of clinical relevance. To address this engineering hurdle, we sought to em...

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Bibliographic Details
Main Authors: Mark J. Osborn, Christopher J. Lees, Amber N. McElroy, Sarah C. Merkel, Cindy R. Eide, Wendy Mathews, Colby J. Feser, Madison Tschann, Ron T. McElmury, Beau R. Webber, Chong Jai Kim, Bruce R. Blazar, Jakub Tolar
Format: Article
Language:English
Published: MDPI AG 2018-03-01
Series:International Journal of Molecular Sciences
Subjects:
CRISPR/Cas9
recessive dystrophic epidermolysis bullosa
transcriptional activation
homology directed repair
adeno-associated virus
ubiquitous chromatin opening element
T-cells
cord blood
Online Access:http://www.mdpi.com/1422-0067/19/4/946

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http://www.mdpi.com/1422-0067/19/4/946

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