Clinical Medicine of China | 2019
Progress in Gene Therapy of Hereditary Neurological Ion Channel Disease
Abstract
Current therapies for hereditary neuropathy are often difficult to control or have low patient tolerance, so it is imperative to explore new therapies.Gene therapy has been shown that in many studies it may be applied to nervous system diseases and may become an effective treatment for hereditary nervous system ion channel diseases in the future.At the same time, the continuous progress of photogenetics, chemical genetics and gene editing technology also provides a good basis for the development of gene therapy for hereditary neuropathy.We will review the methods, mechanisms, transduction vectors, promoters and administration routes of gene therapy for hereditary neurological ion channel disease. \n \n \nKey words: \nIon channel disease;\xa0Gene therapy;\xa0Gene therapy;\xa0Gene editing;\xa0CRISPR/Cas9