Progress in RNA-targeting and Gene Editing Therapies for Transthyretin Amyloidosis Cardiomyopathy

Progress in RNA-targeting and Gene Editing Therapies for Transthyretin Amyloidosis Cardiomyopathy

Transthyretin(TTR) protein is a tetramer protein, synthesized mainly by the liver. TTR can be misfolded and deposited as amyloid fibrilae and deposited in the myocardial interstroma leading to transthyroxin amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM was included in China's First List of Rare...

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Bibliographic Details
Main Authors: NIU Ziran, HU Yang, LIU Qingyang, MA Yuanyuan, LIU Jianing, LIU Xin, ZHANG Bo
Format: Article
Language:zho
Published: Editorial Office of Journal of Rare Diseases 2023-01-01
Series:罕见病研究
Subjects:
ransthyretin amyloidosis caridomyopathy
sirna
antisense oligonucleotide
gene-editing
Online Access:https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2023.01.013

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https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2023.01.013

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