[Antisense therapies for neurological diseases].
Sign Up to like & getrecommendations! Published in 2019 at "Der Nervenarzt"
DOI: 10.1007/s00115-019-0724-4
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… read more here.
Keywords: medicine; neurological diseases; antisense therapies; therapies neurological ... See more keywords
Editorial of the Special Issue: Antisense Therapies
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DOI: 10.3390/biomedicines6040095
Abstract: In this special issue of Biomedicines focusing on Antisense Therapies, we include insightful reviews on the state-of-the-art in relation to a number of specific diseases (in particular, Duchenne muscular dystrophy (DMD) [1–3], amyotrophic lateral sclerosis… read more here.
Keywords: disease; applicability; special issue; aos ... See more keywords
Polyglutamine Ataxias: Our Current Molecular Understanding and What the Future Holds for Antisense Therapies
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DOI: 10.3390/biomedicines9111499
Abstract: Polyglutamine (polyQ) ataxias are a heterogenous group of neurological disorders all caused by an expanded CAG trinucleotide repeat located in the coding region of each unique causative gene. To date, polyQ ataxias encompass six disorders:… read more here.
Keywords: polyglutamine ataxias; ataxias current; current molecular; understanding future ... See more keywords