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The potential of CRISPR/Cas9 genome editing for the study and treatment of intervertebral disc pathologies

The CRISPR/Cas9 system has emerged as a powerful tool for mammalian genome engineering. In basic and translational intervertebral disc (IVD) research, this technique has remarkable potential to answer fundamental questions on pathway interactions, to simulate IVD pathologies, and to promote drug dev...

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Detalles Bibliográficos
Autores principales: Krupkova, Olga, Cambria, Elena, Besse, Lenka, Besse, Andrej, Bowles, Robert, Wuertz‐Kozak, Karin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6686831/
https://www.ncbi.nlm.nih.gov/pubmed/31463435
http://dx.doi.org/10.1002/jsp2.1003
Descripción
Sumario:The CRISPR/Cas9 system has emerged as a powerful tool for mammalian genome engineering. In basic and translational intervertebral disc (IVD) research, this technique has remarkable potential to answer fundamental questions on pathway interactions, to simulate IVD pathologies, and to promote drug development. Furthermore, the precisely targeted CRISPR/Cas9 gene therapy holds promise for the effective and targeted treatment of degenerative disc disease and low back pain. In this perspective, we provide an overview of recent CRISPR/Cas9 advances stemming from/with transferability to IVD research, outline possible treatment approaches for degenerative disc disease, and discuss current limitations that may hinder clinical translation.