Antisense Oligonucleotides as a Potential Treatment for Brain Deficits Observed in Myotonic Dystrophy Type 1

By
Justin.choi
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0
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The authors indicated that the IONIS 486178 antisense oligonucleotide (ASO) targeted mutant DMPK mRNAs in the brain and strongly supported the feasibility of a therapy for dystrophia myotonica type 1 patients based on the intrathecal injection of an ASO.
[Gene Therapy]

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