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Development of NanoBRET cellular target engagement assays in primary neurons for activating mutants of p21-activated kinase 1.

Title: Development of NanoBRET cellular target engagement assays in primary neurons for activating mutants of p21-activated kinase 1.
Authors: Capener JL; Badillo-Martinez A; Awada B; Davis-Gilbert ZW; Kramer TW; Blair CS; Bashore FM; Al-Ali H; Axtman AD
Source: BioRxiv : the preprint server for biology [bioRxiv] 2026 May 06. Date of Electronic Publication: 2026 May 06.
Publication Type: Journal Article; Preprint
Language: English
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
Abstract: The p21-activated kinases (PAKs) are a group of serine-threonine kinases central to multiple signaling pathways that govern cell survival and proliferation. Aberrant activity of PAK1, the most well characterized member of the PAK family, drives progression of several malignancies and brain disorders, including Alzheimer's disease and neurodevelopmental disorders. Despite growing interest in PAK1 as a drug target for these diseases, there is no assay to evaluate the intracellular target engagement of PAK1 inhibitors. To address this need, we developed first-in-class NanoBRET assays for wild-type PAK1 and a neurodevelopmental disorder-causing gain-of-function PAK1 mutant. Furthermore, we executed our novel PAK1 NanoBRET assay to evaluate target engagement of PAK1 inhibitors in primary hippocampal neurons. To the best of our knowledge, this is the first demonstration of a NanoBRET cellular target engagement assay in primary neurons, thereby increasing the relevance of our work by confirming PAK1 inhibitor binding to the aberrant form of the protein in primary neurons.
Entry Date(s): Date Created: 20260518 Date Completed: 20260518 Latest Revision: 20260518
Update Code: 20260518
PubMed Central ID: PMC13174495
DOI: 10.64898/2026.05.03.722513
PMID: 42146347
Database: MEDLINE

Journal Article; Preprint