Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis
# Background Amyloidosis is a group of diseases with the common pathophysiology of protein misfolding and aberrant deposition in tissue. There are both acquired and hereditary forms of this disease, and this review focuses on the latter hereditary transthyretin-mediated (hATTR). hATTR affects about...
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| Format: | Article |
| Language: | English |
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Open Medical Publishing
2023-01-01
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| Series: | Health Psychology Research |
| Online Access: | https://doi.org/10.52965/001c.67910 |
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| author | Christopher Robinson Cynthia Pham Alec M. Zamarripa Chase S. Dugay Christopher A. Lee Amnon A. Berger Avi Landman Elyse M. Cornett Hisham Kassem Alan D. Kaye Ivan Urits Omar Viswanath Latha Ganti |
| author_facet | Christopher Robinson Cynthia Pham Alec M. Zamarripa Chase S. Dugay Christopher A. Lee Amnon A. Berger Avi Landman Elyse M. Cornett Hisham Kassem Alan D. Kaye Ivan Urits Omar Viswanath Latha Ganti |
| author_sort | Christopher Robinson |
| collection | DOAJ |
| description | # Background
Amyloidosis is a group of diseases with the common pathophysiology of protein misfolding and aberrant deposition in tissue. There are both acquired and hereditary forms of this disease, and this review focuses on the latter hereditary transthyretin-mediated (hATTR). hATTR affects about 50,000 individuals globally and mostly appears as one of three syndromes - cardiac, polyneuropathy, and oculoleptomeningeal. Polyneuropathy is the most common form, and there is usually some overlap in individual patients.
# Results
Recently, novel therapeutic options emerged in the form of groundbreaking drugs, Patisiran and Inotersen, small interfering RNA molecules that target TTR and reduce the production of this protein. By targeting TTR mRNA transcripts, Inotersen decreases protein translation and production, reducing the deposition of misfolded proteins. It was shown to be both effective and safe for use and specifically formulated to concentrate in the liver -- where protein production takes place.
# Conclusion
hATTR is a rare, progressive, and debilitating disease. Its most common presentation is that of polyneuropathy, and it carries a very poor prognosis and a natural history conveying a median survival of \< 12 years. Novel therapeutic options are groundbreaking by providing disease-modifying specific, targeted therapies against TTR production and deposition. The use of RNA interference (RNAi) opens the door to the treatment of hereditary diseases by targeting them at the genetic level. |
| format | Article |
| id | doaj-art-9cbad0f2f0034dc993321406e418dd81 |
| institution | Kabale University |
| issn | 2420-8124 |
| language | English |
| publishDate | 2023-01-01 |
| publisher | Open Medical Publishing |
| record_format | Article |
| series | Health Psychology Research |
| spelling | doaj-art-9cbad0f2f0034dc993321406e418dd812025-02-11T20:30:38ZengOpen Medical PublishingHealth Psychology Research2420-81242023-01-01105Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) AmyloidosisChristopher RobinsonCynthia PhamAlec M. ZamarripaChase S. DugayChristopher A. LeeAmnon A. BergerAvi LandmanElyse M. CornettHisham KassemAlan D. KayeIvan UritsOmar ViswanathLatha Ganti# Background Amyloidosis is a group of diseases with the common pathophysiology of protein misfolding and aberrant deposition in tissue. There are both acquired and hereditary forms of this disease, and this review focuses on the latter hereditary transthyretin-mediated (hATTR). hATTR affects about 50,000 individuals globally and mostly appears as one of three syndromes - cardiac, polyneuropathy, and oculoleptomeningeal. Polyneuropathy is the most common form, and there is usually some overlap in individual patients. # Results Recently, novel therapeutic options emerged in the form of groundbreaking drugs, Patisiran and Inotersen, small interfering RNA molecules that target TTR and reduce the production of this protein. By targeting TTR mRNA transcripts, Inotersen decreases protein translation and production, reducing the deposition of misfolded proteins. It was shown to be both effective and safe for use and specifically formulated to concentrate in the liver -- where protein production takes place. # Conclusion hATTR is a rare, progressive, and debilitating disease. Its most common presentation is that of polyneuropathy, and it carries a very poor prognosis and a natural history conveying a median survival of \< 12 years. Novel therapeutic options are groundbreaking by providing disease-modifying specific, targeted therapies against TTR production and deposition. The use of RNA interference (RNAi) opens the door to the treatment of hereditary diseases by targeting them at the genetic level.https://doi.org/10.52965/001c.67910 |
| spellingShingle | Christopher Robinson Cynthia Pham Alec M. Zamarripa Chase S. Dugay Christopher A. Lee Amnon A. Berger Avi Landman Elyse M. Cornett Hisham Kassem Alan D. Kaye Ivan Urits Omar Viswanath Latha Ganti Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis Health Psychology Research |
| title | Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis |
| title_full | Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis |
| title_fullStr | Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis |
| title_full_unstemmed | Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis |
| title_short | Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis |
| title_sort | inotersen to treat polyneuropathy associated with hereditary transthyretin hattr amyloidosis |
| url | https://doi.org/10.52965/001c.67910 |
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