دورية أكاديمية
PNPLA3 as a therapeutic target for fatty liver disease : the evidence to date
| العنوان: | PNPLA3 as a therapeutic target for fatty liver disease : the evidence to date |
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| المؤلفون: | Alessandro Cherubini, Elia Casirati, Melissa Tomasi, Luca Valenti |
| المساهمون: | A. Cherubini, E. Casirati, M. Tomasi, L.V.C. Valenti |
| بيانات النشر: | Taylor & Francis |
| سنة النشر: | 2021 |
| المجموعة: | The University of Milan: Archivio Istituzionale della Ricerca (AIR) |
| مصطلحات موضوعية: | PNPLA3, HSD17B13, Precision medicine, Nonalcoholic fatty liver disease, Liver organoids, Settore MED/03 - Genetica Medica |
| الوصف: | Introduction An interaction between metabolic triggers and inherited predisposition underpins the development and progression of nonalcoholic Fatty Liver disease (NAFLD) and fatty liver disease in general. Among the specific NAFLD risk variants, PNPLA3 rs738409 C>G, encoding for the p.I148M protein variant, accounts for the largest fraction of liver disease heritability and is being intensively scrutinized. It promotes intrahepatic lipid accumulation and is associated with lipotoxicity and the more severe phenotypes, including fibrosis and carcinogenesis. Therefore, PNPLA3 appears as an appealing therapeutic target to counter NAFLD progression. Areas covered The scope of this review is to briefly describe the PNPLA3 gene and protein function before discussing therapeutic approaches for fatty liver aiming at this target. Literature review was carried out searching through PubMed and clinicaltrials.gov website and focusing on the most recent works and reviews. Expert opinion The main therapeutic strategies under development for NAFLD have shown variable efficacy and side-effects likely due to disease heterogeneity and lack of engagement of the main pathogenic drivers of liver disease. To overcome these limitations, new strategies are becoming available for targeting PNPLA3 p.I148M, responsible for a large fraction of disease susceptibility. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | info:eu-repo/semantics/altIdentifier/pmid/34904923; info:eu-repo/semantics/altIdentifier/wos/WOS:000734219000001; journal:EXPERT OPINION ON THERAPEUTIC TARGETS; http://hdl.handle.net/2434/889573Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85121834172 |
| DOI: | 10.1080/14728222.2021.2018418 |
| الإتاحة: | https://doi.org/10.1080/14728222.2021.2018418Test http://hdl.handle.net/2434/889573Test |
| حقوق: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.641A26CC |
| قاعدة البيانات: | BASE |
| DOI: | 10.1080/14728222.2021.2018418 |
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