Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases
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DOI: 10.3390/pharmaceutics14081708
Abstract: Antisense oligonucleotides (ASOs) are single-stranded nucleic acid strings that can be used to selectively modify protein synthesis by binding complementary (pre-)mRNA sequences. By specific arrangements of DNA and RNA into a chain of nucleic acids… read more here.
Keywords: toxic gain; personalized allele; gain function; allele specific ... See more keywords