title: Oligonucleotide-based therapy for neurodegenerative diseases.
abstract: Molecular genetics insight into the pathogenesis of several neurodegenerative diseases, such as Alzheimer׳s disease, Parkinson׳s disease, Huntington׳s disease and amyotrophic lateral sclerosis, encourages direct interference with the activity of neurotoxic genes or the molecular activation of neuroprotective pathways. Oligonucleotide-based therapies are recently emerging as an efficient strategy for drug development and these can be employed as new treatments of neurodegenerative states. Here we review advances in this field in recent years which suggest an encouraging assessment that oligonucleotide technologies for targeting of RNAs will enable the development of new therapies and will contribute to preservation of brain integrity.
authors.affiliation: Department of Molecular Genetics, Faculty of Biochemistry, Weizmann Institute of Science, Rehovot, Israel. Electronic address: iddo.magen@weizmann.ac.il.
authors.forename: Iddo
authors.initials: I
authors.lastname: Magen
authors.affiliation: Department of Molecular Genetics, Faculty of Biochemistry, Weizmann Institute of Science, Rehovot, Israel. Electronic address: eran.hornstein@weizmann.ac.il.
authors.forename: Eran
authors.initials: E
authors.lastname: Hornstein
mesh_terms: D000818:Animals; D001921:Brain / Q000187:drug effects; D006801:Humans; D035683:MicroRNAs / Q000187:drug effects / Q000627:therapeutic use; D058990:Molecular Targeted Therapy / Q000379:methods; D019636:Neurodegenerative Diseases / Q000188:drug therapy*; D016376:Oligonucleotides, Antisense / Q000008:administration & dosage / Q000627:therapeutic use*; D034622:RNA Interference; D034743:RNA-Induced Silencing Complex
chemical_list: D035683:MicroRNAs; D016376:Oligonucleotides, Antisense; D034743:RNA-Induced Silencing Complex
keywords: Antisense oligonucleotides; Delivery; Neurodegeneration; RNA interference; microRNA; siRNA