Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review
Transthyretin amyloidosis (ATTR) manifests as wild-type (ATTRwt) and hereditary/mutant (ATTRv) forms and can lead to heart failure due to cardiac amyloidopathy. Diagnosing ATTR, particularly in asymptomatic carriers of pathogenic variants, remains challenging despite the advances. Complex and multi-...
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| Format: | Article |
| Language: | English |
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Medical University of Gdańsk
2024-06-01
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| Series: | European Journal of Translational and Clinical Medicine |
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| Online Access: | https://ejtcm.gumed.edu.pl/articles/189121.pdf |
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| author | Maria Kulak Miriam Lang Natalia Lange |
| author_facet | Maria Kulak Miriam Lang Natalia Lange |
| author_sort | Maria Kulak |
| collection | DOAJ |
| description | Transthyretin amyloidosis (ATTR) manifests as wild-type (ATTRwt) and hereditary/mutant (ATTRv) forms and can lead to heart failure due to cardiac amyloidopathy. Diagnosing ATTR, particularly in asymptomatic carriers of pathogenic variants, remains challenging despite the advances. Complex and multi-aspect management involves a limited range of well-examined conventional therapies to address the heart failure and frequently coexisting arrhythmias and valvular issues. Disease-modifying treatment, RNA-based treatments, CRISPR-Cas9 gene editing and monoclonal antibodies targeting amyloid deposits are recent and promising innovations. This review explores the diagnostic intricacies, therapeutic dilemmas and emerging solutions in ATTR cardiomyopathy. The significance of early detection and precise, targeted approaches to enhance patient outcomes is underscored. |
| format | Article |
| id | doaj-art-bcf18905754549a2b64a5a4b835eb95e |
| institution | Kabale University |
| issn | 2657-3148 2657-3156 |
| language | English |
| publishDate | 2024-06-01 |
| publisher | Medical University of Gdańsk |
| record_format | Article |
| series | European Journal of Translational and Clinical Medicine |
| spelling | doaj-art-bcf18905754549a2b64a5a4b835eb95e2025-08-20T03:59:52ZengMedical University of GdańskEuropean Journal of Translational and Clinical Medicine2657-31482657-31562024-06-01716378189121Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a reviewMaria Kulak0https://orcid.org/0009-0000-6359-0560Miriam Lang1https://orcid.org/0009-0002-5226-1467Natalia Lange2https://orcid.org/0000-0002-1710-4637Faculty of Medicine, Medical University of Gdańsk, PolandLower Silesian Oncology Center, Wrocław, PolandT. Marciniak Memorial Lower Silesian Specialist Hospital, Wrocław, PolandTransthyretin amyloidosis (ATTR) manifests as wild-type (ATTRwt) and hereditary/mutant (ATTRv) forms and can lead to heart failure due to cardiac amyloidopathy. Diagnosing ATTR, particularly in asymptomatic carriers of pathogenic variants, remains challenging despite the advances. Complex and multi-aspect management involves a limited range of well-examined conventional therapies to address the heart failure and frequently coexisting arrhythmias and valvular issues. Disease-modifying treatment, RNA-based treatments, CRISPR-Cas9 gene editing and monoclonal antibodies targeting amyloid deposits are recent and promising innovations. This review explores the diagnostic intricacies, therapeutic dilemmas and emerging solutions in ATTR cardiomyopathy. The significance of early detection and precise, targeted approaches to enhance patient outcomes is underscored.https://ejtcm.gumed.edu.pl/articles/189121.pdfcardiomyopathyamyloidosistransthyretin |
| spellingShingle | Maria Kulak Miriam Lang Natalia Lange Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review European Journal of Translational and Clinical Medicine cardiomyopathy amyloidosis transthyretin |
| title | Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review |
| title_full | Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review |
| title_fullStr | Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review |
| title_full_unstemmed | Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review |
| title_short | Advances in understanding, diagnosis and targeting ATTR cardiomyopathy: a review |
| title_sort | advances in understanding diagnosis and targeting attr cardiomyopathy a review |
| topic | cardiomyopathy amyloidosis transthyretin |
| url | https://ejtcm.gumed.edu.pl/articles/189121.pdf |
| work_keys_str_mv | AT mariakulak advancesinunderstandingdiagnosisandtargetingattrcardiomyopathyareview AT miriamlang advancesinunderstandingdiagnosisandtargetingattrcardiomyopathyareview AT natalialange advancesinunderstandingdiagnosisandtargetingattrcardiomyopathyareview |