Transthyretin Amyloidosis Therapies: Guiding the Future
Transthyretin (TTR) amyloidosis (ATTR) is a progressive condition characterized by multiorgan accumulation of amyloid deposits composed of transthyretin (TTR) fibrils. Over the past decades, despite being a rare disease, ATTR amyloidosis has enabled top-tier therapeutics. In the 90s, organ transplan...
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| Format: | Article |
| Language: | English |
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Permanyer
2021-01-01
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| Series: | Revista de Investigación Clínica |
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| Online Access: | https://www.clinicalandtranslationalinvestigation.com/frame_esp.php?id=400 |
| _version_ | 1798017172577976320 |
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| author | Alejandra González-Duarte |
| author_facet | Alejandra González-Duarte |
| author_sort | Alejandra González-Duarte |
| collection | DOAJ |
| description | Transthyretin (TTR) amyloidosis (ATTR) is a progressive condition characterized by multiorgan accumulation of amyloid deposits composed of transthyretin (TTR) fibrils. Over the past decades, despite being a rare disease, ATTR amyloidosis has enabled top-tier therapeutics. In the 90s, organ transplantation was the mainstream therapeutic option and fostered distinct approaches, such as combined liver-heart transplant and domino (sequential) liver transplantation. Likewise, several TTR molecule stabilizers were developed successfully. Over the past decade, oriented genetic therapies emerged to prevent, control, and, surprisingly, reverse amyloid deposition. Silencing the TTR gene using different strategies is flourishing, and ongoing trials continue to evaluate diverse approaches to optimize their application. The following perspective describes the currently available treatments for ATTR amyloidosis and the prospects on the potential application of these strategies in other medical fields. ( |
| first_indexed | 2024-04-11T16:01:32Z |
| format | Article |
| id | doaj.art-7c14845efc204c6cb4357703126d3ee4 |
| institution | Directory Open Access Journal |
| issn | 0034-8376 2564-8896 |
| language | English |
| last_indexed | 2024-04-11T16:01:32Z |
| publishDate | 2021-01-01 |
| publisher | Permanyer |
| record_format | Article |
| series | Revista de Investigación Clínica |
| spelling | doaj.art-7c14845efc204c6cb4357703126d3ee42022-12-22T04:14:56ZengPermanyerRevista de Investigación Clínica0034-83762564-88962021-01-0173510.24875/RIC.21000323Transthyretin Amyloidosis Therapies: Guiding the FutureAlejandra González-Duarte0Department of Neurology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, MexicoTransthyretin (TTR) amyloidosis (ATTR) is a progressive condition characterized by multiorgan accumulation of amyloid deposits composed of transthyretin (TTR) fibrils. Over the past decades, despite being a rare disease, ATTR amyloidosis has enabled top-tier therapeutics. In the 90s, organ transplantation was the mainstream therapeutic option and fostered distinct approaches, such as combined liver-heart transplant and domino (sequential) liver transplantation. Likewise, several TTR molecule stabilizers were developed successfully. Over the past decade, oriented genetic therapies emerged to prevent, control, and, surprisingly, reverse amyloid deposition. Silencing the TTR gene using different strategies is flourishing, and ongoing trials continue to evaluate diverse approaches to optimize their application. The following perspective describes the currently available treatments for ATTR amyloidosis and the prospects on the potential application of these strategies in other medical fields. (https://www.clinicalandtranslationalinvestigation.com/frame_esp.php?id=400Transthyretin amyloidosis. Hereditary amyloidosis. siRNA. Lipid nanoparticles. |
| spellingShingle | Alejandra González-Duarte Transthyretin Amyloidosis Therapies: Guiding the Future Revista de Investigación Clínica Transthyretin amyloidosis. Hereditary amyloidosis. siRNA. Lipid nanoparticles. |
| title | Transthyretin Amyloidosis Therapies: Guiding the Future |
| title_full | Transthyretin Amyloidosis Therapies: Guiding the Future |
| title_fullStr | Transthyretin Amyloidosis Therapies: Guiding the Future |
| title_full_unstemmed | Transthyretin Amyloidosis Therapies: Guiding the Future |
| title_short | Transthyretin Amyloidosis Therapies: Guiding the Future |
| title_sort | transthyretin amyloidosis therapies guiding the future |
| topic | Transthyretin amyloidosis. Hereditary amyloidosis. siRNA. Lipid nanoparticles. |
| url | https://www.clinicalandtranslationalinvestigation.com/frame_esp.php?id=400 |
| work_keys_str_mv | AT alejandragonzalezduarte transthyretinamyloidosistherapiesguidingthefuture |