Search

Your search keyword '"Rajarathinam, Thenmozhi"' showing total 9 results
Start Over Author "Rajarathinam, Thenmozhi" Search Limiters Full Text
9 results on '"Rajarathinam, Thenmozhi"'

7. Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy

Author

Byung-Ok Choi, Rajarathinam Thenmozhi, Na Young Park, Young Bin Hong, and Ji-Su Lee

Subjects
0301 basic medicine, Genetic enhancement, Mutant, Inheritance (genetic algorithm), Inherited Peripheral Neuropathy, Review Article, Gene editing, Biology, Bioinformatics, 03 medical and health sciences, Cellular and Molecular Neuroscience, Gene therapy, 030104 developmental biology, 0302 clinical medicine, Genome editing, microRNA, Antisense oligonucleotide, Neurology (clinical), Inherited peripheral neuropathy, Gene, 030217 neurology & neurosurgery, Function (biology), miRNA
Abstract

Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small molecules. However, gene therapy-based therapeutic strategies have not been aggressively investigated, likely due to the complexities of inheritance in IPN. Indeed, because the majority of the causative mutations of IPN lead to gainof- function rather than loss-of-function, developing a therapeutic strategy is more difficult, especially considering gene therapy for genetic diseases began with the simple idea of replacing a defective gene with a functional copy. Recent advances in gene manipulation technology have brought novel approaches to gene therapy and its clinical application for IPN treatment. For example, in addition to the classically used gene replacement for mutant genes in recessively inherited IPN, other techniques including gene addition to modify the disease phenotype, modulations of target gene expression, and techniques to edit mutant genes have been developed and evaluated as potent therapeutic strategies for dominantly inherited IPN. In this review, the current status of gene therapy for IPN and future perspectives will be discussed.

Published
2020

Catalog

Books, media, physical & digital resources
See catalog results