[Antisense therapies for neurological diseases].
[Antisense therapies for neurological diseases].
Nervenarzt. 2019 Jun 04;:
Authors: Pulst SM
Abstract
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.
PMID: 31165208 [PubMed - as supplied by publisher]
Source: Der Nervenarzt - Category: Neurology Authors: Pulst SM Tags: Nervenarzt Source Type: research
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