Single-cell proteo-genomic reveals a comprehensive map of centrosome-associated spliceosome components

Cerulo, Luigi; Pezzella, Nunziana; Caruso, Francesca Pia; Parente, Paola; Remo, Andrea; Giordano, Guido; Forte, Nicola; Busselez, Johan; Boschi, Federico; Galiè, Mirco; Bazzoli, Franco; Pancione, Massimo

doi:10.1016/j.isci.2023.106602

Cited by 2 publications

(1 citation statement)

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“…For example, eupatilin, a drug used to treat gastritis and peptic ulcers, was identified as a positive hit that could induce ciliogenesis in a drug screening using CEP290-null cells ( Kim et al, 2018 ). Recent studies have also revealed that primary cilia are involved in a wide variety of cellular functions such as the regulation of spliceosome and the facilitation of signaling ( Cerulo et al, 2023 ; Macarelli et al, 2023 ), suggesting that chemicals targeting primary cilia may be applicable to treat an array of diseases than previously thought. To date, multiple examples of ciliopathy IRD gene therapy in large animal models of retinal degeneration have shown that ciliopathy phenotypes may be reversed.…”

Section: Discussionmentioning

confidence: 99%

Recent advances in the understanding of cilia mechanisms and their applications as therapeutic targets

Saito,

Otsu,

Miyadera

et al. 2023

Front. Mol. Biosci.

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The primary cilium is a single immotile microtubule-based organelle that protrudes into the extracellular space. Malformations and dysfunctions of the cilia have been associated with various forms of syndromic and non-syndromic diseases, termed ciliopathies. The primary cilium is therefore gaining attention due to its potential as a therapeutic target. In this review, we examine ciliary receptors, ciliogenesis, and ciliary trafficking as possible therapeutic targets. We first discuss the mechanisms of selective distribution, signal transduction, and physiological roles of ciliary receptors. Next, pathways that regulate ciliogenesis, specifically the Aurora A kinase, mammalian target of rapamycin, and ubiquitin-proteasome pathways are examined as therapeutic targets to regulate ciliogenesis. Then, in the photoreceptors, the mechanism of ciliary trafficking which takes place at the transition zone involving the ciliary membrane proteins is reviewed. Finally, some of the current therapeutic advancements highlighting the role of large animal models of photoreceptor ciliopathy are discussed.

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Section: Discussionmentioning

confidence: 99%