Azithromycin induces read-through of the nonsense Apc allele and prevents intestinal tumorigenesis in C3B6F1 Apc/+ mice

Semba, Ryoko; Morioka, Takamitsu; Yanagihara, Hirokazu; Suzuki, Koichi; Tachibana, Hirotaka; Hamoya, Takahiro; Horimoto, Yoshiya; Imaoka, Tatsuhiko; Saito, Mitsue; Kakinuma, Shizuko; Arai, Masami

doi:10.1016/j.biopha.2023.114968

Cited by 4 publications

(1 citation statement)

References 34 publications

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“…Furthermore, treatment with tylosin reduced the tumorigenic effects of APC nonsense mutations in a mouse xenograft model (as determined by a 50% reduction in tumour size compared to control) and increased the lifespan of APC ‐deficient mice 60 . Similarly, azithromycin acted anti‐tumorigenic in a different mouse model for familial adenomatous polyposis by promoting SCR of a nonsense mutation (NM_007462.4:c.2549 T > A; NP_031488.2:p.Leu850Ter) in the APC gene 61 …”

Section: Non‐aminoglycoside Antibioticsmentioning

confidence: 99%

Stop codon readthrough as a treatment option for epidermolysis bullosa—Where we are and where we are going

Zandanell,

Wießner,

Bauer

et al. 2024

Experimental Dermatology

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In the context of rare genetic diseases caused by nonsense mutations, the concept of induced stop codon readthrough (SCR) represents an attractive avenue in the ongoing search for improved treatment options. Epidermolysis bullosa (EB)—exemplary for this group of diseases—describes a diverse group of rare, blistering genodermatoses. Characterized by extreme skin fragility upon minor mechanical trauma, the most severe forms often result from nonsense mutations that lead to premature translation termination and loss of function of essential proteins at the dermo‐epidermal junction. Since no curative interventions are currently available, medical care is mainly limited to alleviating symptoms and preventing complications. Complementary to attempts of gene, cell and protein therapy in EB, SCR represents a promising medical alternative. While gentamicin has already been examined in several clinical trials involving EB, other potent SCR inducers, such as ataluren, may also show promise in treating the hitherto non‐curative disease. In addition to the extensively studied aminoglycosides and their derivatives, several other substance classes—non‐aminoglycoside antibiotics and non‐aminoglycoside compounds—are currently under investigation. The extensive data gathered in numerous in vitro experiments and the perspectives they reveal in the clinical setting will be discussed in this review.

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Section: Non‐aminoglycoside Antibioticsmentioning

confidence: 99%