Using Transcriptomic Analysis to Assess Double-Strand Break Repair Activity: Towards Precise in Vivo Genome Editing

Using Transcriptomic Analysis to Assess Double-Strand Break Repair Activity: Towards Precise in Vivo Genome Editing

Mutations in more than 200 retina-specific genes have been associated with inherited retinal diseases. Genome editing represents a promising emerging field in the treatment of monogenic disorders, as it aims to correct disease-causing mutations within the genome. Genome editing relies on highly spec...

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Bibliographic Details
Main Authors: Giovanni Pasquini, Virginia Cora, Anka Swiersy, Kevin Achberger, Lena Antkowiak, Brigitte Müller, Tobias Wimmer, Sabine Anne-Kristin Fraschka, Nicolas Casadei, Marius Ueffing, Stefan Liebau, Knut Stieger, Volker Busskamp
Format: Article
Language:English
Published: MDPI AG 2020-02-01
Series:International Journal of Molecular Sciences
Subjects:
inherited retinal dystrophies
double-strand breaks
dsb repair pathways
single-cell rna-seq
hipsc-derived retinal organoids
photoreceptors
Online Access:https://www.mdpi.com/1422-0067/21/4/1380

Internet

https://www.mdpi.com/1422-0067/21/4/1380

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