ReviewInternational journal of molecular sciences2021
Small-Molecule Therapeutic Perspectives for the Treatment of Progeria.
Review in International journal of molecular sciences, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
15 citing papers in PubMed, 22 citations in OpenAlex.
- First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- The nuclear envelope and nuclear pore complexes in neurodegenerative diseases.Frontiers in cell and developmental biology · 2025Review
- The Dental and Oral Significance of Hutchinson-Gilford Prfogeria Syndrome : The Dental and Oral Significance of Hutchinson-Gilford Prfogeria Syndrome.Galen medical journal · 2025Review
- The secretome atlas of two mouse models of progeria.Aging cell · 2023Article
- [Research advances in pharmacotherapy for rare diseases in children].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2023Review
- Article
- New Pharmacological Approaches for Rare Diseases.International journal of molecular sciences · 2023Article
- Discovery of a new hereditary RECQ helicase disorder RECON syndrome positions the replication stress response and genome homeostasis as centrally important processes in aging and age-related disease.Ageing research reviews · 2023Review
- Molecular and therapeutic insights of rapamycin: a multi-faceted drug from Streptomyces hygroscopicus.Molecular biology reports · 2023Review
- Mechanistic insight into impact of phosphorylation on the enzymatic steps of farnesyltransferase.Protein science : a publication of the Protein Society · 2022Article
- Progeria and Aging-Omics Based Comparative Analysis.Biomedicines · 2022Article
- SerpinE1 drives a cell-autonomous pathogenic signaling in Hutchinson-Gilford progeria syndrome.Cell death & disease · 2022Article
- New Trends in Aging Drug Discovery.Biomedicines · 2022Review
- Clinical manifestations and gene analysis of Hutchinson-Gilford progeria syndrome: A case report.World journal of clinical cases · 2022Article
- Progerin-Induced Impairment in Wound Healing and Proliferation in Vascular Endothelial Cells.Frontiers in aging · 2022Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Hutchinson-Gilford progeria syndrome (HGPS), or progeria, is an extremely rare disorder that belongs to the class of laminopathies, diseases characterized by alterations in the genes that encode for the lamin proteins or for their associated interacting proteins. In particular, progeria is caused by a point mutation in the gene that codifies for the lamin A gene. This mutation ultimately leads to the biosynthesis of a mutated version of lamin A called progerin, which accumulates abnormally in the nuclear lamina. This accumulation elicits several alterations at the nuclear, cellular, and tissue levels that are phenotypically reflected in a systemic disorder with important alterations, mainly in the cardiovascular system, bones, skin, and overall growth, which results in premature death at an average age of 14.5 years. In 2020, lonafarnib became the first (and only) FDA approved drug for treating progeria. In this context, the present review focuses on the different therapeutic strategies currently under development, with special attention to the new small molecules described in recent years, which may represent the upcoming first-in-class drugs with new mechanisms of action endowed with effectiveness not only to treat but also to cure progeria.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.