Article
Pmp22 mutant allele-specific siRNA alleviates demyelinating neuropathic phenotype in vivo.
Neurobiology of disease - 1 Apr 2017
Lee Ji-Su, Chang Eun Hyuk, Koo Ok Jae, Jwa Dong Hwan, Mo Won Min, Kwak Geon, Moon Hyo Won, Park Hwan Tae, Hong Young Bin, Choi Byung-Ok
Abstract excerpt
Charcot-Marie-Tooth disease (CMT) is a genetic disorder that can be caused by aberrations in >80 genes. CMT has heterogeneous modes of inheritance, including autosomal dominant, autosomal recessive, X-linked dominant, and X-linked recessive. Over 95% of cases are dominantly inherited. In this study, we investigated whether regulation of a mutant allele by an allele-specific small interfering RNA (siRNA) can...
Topics
- Alleles
- Animals
- Charcot-Marie-Tooth Disease
- Demyelinating Diseases
- Mice, Transgenic
- Mutation
- Myelin Proteins
- Phenotype
- RNA, Small Interfering
- Schwann Cells
- Sciatic Nerve