Treatment of ATTR cardiomyopathy with a TTR specific antisense oligonucleotide, inotersen.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS(2019)

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摘要
Transthyretin (TTR) cardiomyopathy can occur as the result of an inherited mutation in transthyretin (ATTRm) or due to deposition of normal transthyretin in older individuals (ATTRWT). TTR cardiomy...
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关键词
specific antisense oligonucleotide,attr
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