Article
Predictable CRISPR/Cas9-Mediated COL7A1 Reframing for Dystrophic Epidermolysis Bullosa.
The Journal of investigative dermatology - 1 Oct 2020
Kocher Thomas, March Oliver Patrick, Bischof Johannes, Liemberger Bernadette, Hainzl Stefan, Klausegger Alfred, Hoog Anna, Strunk Dirk, Bauer Johann Wolfgang, Koller Ulrich
Abstract excerpt
End-joining‒based gene editing is frequently used for efficient reframing and knockout of target genes. However, the associated random, unpredictable, and often heterogeneous repair outcomes limit its applicability for therapeutic approaches. This study revealed more precise and predictable outcomes simply on the basis of the sequence context at the CRISPR/Cas9 target site. The severe dystrophic form of the...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
