RNA-targeting and gene editing therapies for transthyretin amyloidosis
RNA-targeting and gene editing therapies for transthyretin amyloidosis
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London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Contents
Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver and secreted into the plasma. TTR molecules can misfold and form amyloid fibrils in the heart and peripheral nerves, either as a result of gene variants in
TTR
or as an ageing-related phenomenon, which can lead to amyloid TTR (ATTR) amyloidosis. Some of the proposed s...
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Full title
RNA-targeting and gene editing therapies for transthyretin amyloidosis
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TN_cdi_proquest_miscellaneous_2642886668
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2642886668
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ISSN
1759-5002
E-ISSN
1759-5010
DOI
10.1038/s41569-022-00683-z