Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis
The disease of transthyretin (TTR) amyloidosis (ATTR) has been known since the 1960s, and during the past 60 or so years, there has been a sustained period of steady discoveries that have led to the current model of ATTR pathogenesis. More recent research has achieved major advances in both diagnost...
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MDPI AG
2024-09-01
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| author | Avi Chakrabartty |
| author_facet | Avi Chakrabartty |
| author_sort | Avi Chakrabartty |
| collection | DOAJ |
| description | The disease of transthyretin (TTR) amyloidosis (ATTR) has been known since the 1960s, and during the past 60 or so years, there has been a sustained period of steady discoveries that have led to the current model of ATTR pathogenesis. More recent research has achieved major advances in both diagnostics and therapeutics for ATTR, which are having a significant impact on ATTR patients today. Aiding these recent achievements has been the remarkable ability of cryo-electron microscopy (EM) to determine high-resolution structures of amyloid fibrils obtained from individual patients. Here, we will examine the cryo-EM structures of transthyretin amyloid fibrils to explore the structural basis of the two monoclonal antibody therapies for ATTR that are in clinical trials, ALXN-2220 and Coramitug, as well as to point out potential applications of this approach to other systemic amyloid diseases. |
| format | Article |
| id | doaj-art-e71e0c31f22f4dd09cdecf9ea4c32fd1 |
| institution | OA Journals |
| issn | 1424-8247 |
| language | English |
| publishDate | 2024-09-01 |
| publisher | MDPI AG |
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| series | Pharmaceuticals |
| spelling | doaj-art-e71e0c31f22f4dd09cdecf9ea4c32fd12025-08-20T01:55:46ZengMDPI AGPharmaceuticals1424-82472024-09-01179122510.3390/ph17091225Structural Basis for Monoclonal Antibody Therapy for Transthyretin AmyloidosisAvi Chakrabartty0Department of Medical Biophysics, University of Toronto, Toronto, ON M5G 2M9, CanadaThe disease of transthyretin (TTR) amyloidosis (ATTR) has been known since the 1960s, and during the past 60 or so years, there has been a sustained period of steady discoveries that have led to the current model of ATTR pathogenesis. More recent research has achieved major advances in both diagnostics and therapeutics for ATTR, which are having a significant impact on ATTR patients today. Aiding these recent achievements has been the remarkable ability of cryo-electron microscopy (EM) to determine high-resolution structures of amyloid fibrils obtained from individual patients. Here, we will examine the cryo-EM structures of transthyretin amyloid fibrils to explore the structural basis of the two monoclonal antibody therapies for ATTR that are in clinical trials, ALXN-2220 and Coramitug, as well as to point out potential applications of this approach to other systemic amyloid diseases.https://www.mdpi.com/1424-8247/17/9/1225transthyretinamyloidosisantibody therapyprotein structurecryo-electron microscopy |
| spellingShingle | Avi Chakrabartty Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis Pharmaceuticals transthyretin amyloidosis antibody therapy protein structure cryo-electron microscopy |
| title | Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis |
| title_full | Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis |
| title_fullStr | Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis |
| title_full_unstemmed | Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis |
| title_short | Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis |
| title_sort | structural basis for monoclonal antibody therapy for transthyretin amyloidosis |
| topic | transthyretin amyloidosis antibody therapy protein structure cryo-electron microscopy |
| url | https://www.mdpi.com/1424-8247/17/9/1225 |
| work_keys_str_mv | AT avichakrabartty structuralbasisformonoclonalantibodytherapyfortransthyretinamyloidosis |