Genetic Therapy for Intervertebral Disc Degeneration.

Affiliation

Roh EJ(1)(2), Darai A(1)(2), Kyung JW(1), Choi H(1), Kwon SY(1), Bhujel B(1)(2), Kim KT(3)(4), Han I(1).
Author information:
(1)Department of Neurosurgery, CHA Bundang Medical Center, CHA University School of Medicine, Seongnam-si 13496, Korea.
(2)Department of Biomedical Science, CHA Bundang Medical Center, CHA University School of Medicine, Seongnam-si 13496, Korea.
(3)School of Medicine, Department of Neurosurgery, Kyungpook National University, Daegu 41944, Korea.
(4)Department of Neurosurgery, Kyungpook National University Hospital, Daegu 41944, Korea.

Abstract

Intervertebral disc (IVD) degeneration can cause chronic lower back pain (LBP), leading to disability. Despite significant advances in the treatment of discogenic LBP, the limitations of current treatments have sparked interest in biological approaches, including growth factor and stem cell injection, as new treatment options for patients with chronic LBP due to IVD degeneration (IVDD). Gene therapy represents exciting new possibilities for IVDD treatment, but treatment is still in its infancy. Literature searches were conducted using PubMed and Google Scholar to provide an overview of the principles and current state of gene therapy for IVDD. Gene transfer to degenerated disc cells in vitro and in animal models is reviewed. In addition, this review describes the use of gene silencing by RNA interference (RNAi) and gene editing by the clustered regularly interspaced short palindromic repeats (CRISPR) system, as well as the mammalian target of rapamycin (mTOR) signaling in vitro and in animal models. Significant technological advances in recent years have opened the door to a new generation of intradiscal gene therapy for the treatment of chronic discogenic LBP.