Investigational agents for autosomal dominant polycystic kidney disease : preclinical and early phase study insights
INTRODUCTION: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited kidney condition caused by a single-gene mutation. It leads patients to kidney failure in more than 50% of cases by the age of 60, and, given the dominant inheritance, this disease is present in the family history in more than 90% of cases.
AREAS COVERED: This review aims to analyze the set of preclinical and early-phase studies to provide a general view of the current progress on ADPKD therapeutic options. Articles from PubMed and the current status of the trials listed in clinicaltrials.gov were examined for the review.
EXPERT OPINION: Many potential therapeutic targets are currently under study for the treatment of ADPKD. A few drugs have reached the clinical phase, while many are currently still in the preclinical phase. Organoids could be a novel approach to the study of drugs in this phase. Other than pharmacological options, very important developing approaches are represented by gene therapy and the use of MiRNA inhibitors.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - year:2024 |
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| Enthalten in: |
Expert opinion on investigational drugs - (2024) vom: 21. Apr., Seite 1-16 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Capelli, Irene [VerfasserIn] |
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| Links: |
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| Themen: |
ADPKD |
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| Anmerkungen: |
Date Revised 21.04.2024 published: Print-Electronic Citation Status Publisher |
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| doi: |
10.1080/13543784.2024.2342327 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM371082463 |
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| 520 | |a INTRODUCTION: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited kidney condition caused by a single-gene mutation. It leads patients to kidney failure in more than 50% of cases by the age of 60, and, given the dominant inheritance, this disease is present in the family history in more than 90% of cases | ||
| 520 | |a AREAS COVERED: This review aims to analyze the set of preclinical and early-phase studies to provide a general view of the current progress on ADPKD therapeutic options. Articles from PubMed and the current status of the trials listed in clinicaltrials.gov were examined for the review | ||
| 520 | |a EXPERT OPINION: Many potential therapeutic targets are currently under study for the treatment of ADPKD. A few drugs have reached the clinical phase, while many are currently still in the preclinical phase. Organoids could be a novel approach to the study of drugs in this phase. Other than pharmacological options, very important developing approaches are represented by gene therapy and the use of MiRNA inhibitors | ||
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| 700 | 1 | |a Berti, Gian Marco |e verfasserin |4 aut | |
| 700 | 1 | |a Aiello, Valeria |e verfasserin |4 aut | |
| 700 | 1 | |a Provenzano, Michele |e verfasserin |4 aut | |
| 700 | 1 | |a La Manna, Gaetano |e verfasserin |4 aut | |
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