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

ABSTRACT: 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.

SUBMITTER: Krupkova O

PROVIDER: S-EPMC6686831 | biostudies-literature | 2018 Mar

REPOSITORIES: biostudies-literature

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

Krupkova Olga O Cambria Elena E Besse Lenka L Besse Andrej A Bowles Robert R Wuertz-Kozak Karin K

JOR spine 20180315 1

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 pr ...[more]

PMID: 31463435

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