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Drug and Gene Therapy for Treating Variant Transthyretin Amyloidosis (ATTRv) Neuropathy
Variant Transthyretin Amyloidosis (ATTRv) neuropathy is an adult-onset, autosomal dominant, lethal, multisystemic disease due to the deposition of mutated transthyretin (TTR) in various organs, commonly involving the peripheral nerves and the heart. Circulating TTR tetramers are unstable due to the...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Bentham Science Publishers
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10207904/ https://www.ncbi.nlm.nih.gov/pubmed/36366846 http://dx.doi.org/10.2174/1570159X21666221108094736 |