Title: | PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models |
Journal : | The Journal of clinical investigation, 128, 1 |
Authors: | Zhao HT, Author ; Damle S ; Ikeda-Lee K ; Kuntz S ; Li J ; Mohan A ; Kim A ; Hung G ; Scheideler MA ; Scherer SS ; Svaren J ; Swayze EE ; Kordasiewicz HB |
Material Type: | Article |
Publication Date: | 2018 |
Size: | p 359 |
Languages: | English |
Keywords : | antisense oligonucleotides ; biological markers ; Charcot-Marie-Tooth disease ; Charcot-Marie-Tooth mouse ; CMT1A ; electrodiagnosis ; gene expression ; motor skills ; myelin protein 22 ; myelination ; nerve conduction velocity ; nervous system histopathology ; PMP22 gene ; preclinical trials ; therapeutic prospects ; transcriptome |
Keywords: | Drug therapy;Gene therapy;Monogenic diseases;Neuroscience;Therapeutics; |
Abstract: |
Comment in:
Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A. [J Clin Invest. 2018] Peripheral neuropathies: Antisense therapy for Charcot-Marie-Tooth disease? [Nat Rev Neurol. 2018] |
Pubmed / DOI : | DOI : 10.1172/JCI96499 / Pubmed : 29202483 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/29202483 |
See also : |