ArticleRSC advances2025
An injectable hydrogel containing
Article in RSC advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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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Authors and funding
10 authors.
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No grant is acknowledged in the PubMed record.
Abstract
Gaucher disease (GD) is a rare inherited genetic disorder resulting from a recessive mutation in the GBA1 gene, which encodes the glucocerebrosidase (GCase) enzyme, resulting in the accumulation of glycolipids within the lysosomes of various body organs. Recently, small molecules that act as stabilizers for mutant proteins have become popular as a potential treatment for genetic disorders. Small-molecule stabilizers improve the stability of mutant proteins by facilitating proper folding, allowing them to regain their functional conformation. This prevents the proteins from entering the proteosomal degradation pathway, ultimately restoring their biological functions. Exploration of novel small-molecule stabilizers of GCase through rational process pipelines using
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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 Socratic graph.