Open AccessAntisense-Therapie neurologischer Erkrankungen
Author(s) -
S. M. Pulst
Publication year - 2019
Publication title -
nervenarzt/der nervenarzt
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.275
H-Index - 44
eISSN - 1433-0407
pISSN - 0028-2804
DOI - 10.1007/s00115-019-0724-4
Subject(s) - medicine , spinal muscular atrophy , disease , antisense therapy , bioinformatics , oligonucleotide , neuroscience , computational biology , gene , pathology , biology , genetics , locked nucleic acid
Despite identification of many genes causing neurodegenerative diseases in the last decades, development of disease-modifying treatments has been slow. Antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy, Duchenne muscular dystrophy and transthyretin amyloidosis predict a robust future for ASOs in medicine. Perhaps the most significant advantage of ASO therapeutics over other small molecule approaches is that acquisition of the target sequence provides immediate knowledge of possible complementary oligonucleotide therapeutics. This review article describes the various types of ASOs, their therapeutic use and the current preclinical efforts to develop new ASO treatments.