Calvin Joel Gordon, Simon Maximus Walker, Julia Christine LeCher, Franck Amblard, Raymond Felix Schinazi, Matthias Götte
{"title":"活性三磷酸形式2′-α-氟,2′-β-溴化吡啶抑制黄热病病毒RNA依赖性RNA聚合酶的机制","authors":"Calvin Joel Gordon, Simon Maximus Walker, Julia Christine LeCher, Franck Amblard, Raymond Felix Schinazi, Matthias Götte","doi":"10.1021/acsinfecdis.5c00086","DOIUrl":null,"url":null,"abstract":"<p><p>Yellow fever virus (YFV) is a deadly mosquito-borne flavivirus that infects approximately 200,000 individuals each year. YFV outbreak and infection are persistent throughout South America and Africa, demonstrating significant epidemic potential. Although an approved and effective vaccine exists, the zoonotic nature of YFV undermines any potential eradication efforts, highlighting the need for effective, direct-acting antivirals. Essential for viral replication, RNA-dependent RNA polymerase (RdRp) is a proven therapeutic target. A prominent example includes sofosbuvir, a 2'-α-fluoro,2'-β-methyluridine prodrug approved for the treatment of hepatitis C virus (HCV), that has demonstrated efficacy against YFV <i>in vitro</i>. A structurally similar 2'-α-fluoro, 2'-β-bromouridine prodrug has exhibited potent anti-YFV activity both <i>in vitro</i> and <i>in vivo</i>. Here, we expressed the full-length nonstructural protein 5 from YFV in insect cells to investigate the active triphosphate form of these prodrugs. Enzyme kinetics indicate that both nucleotide analogs are incorporated less efficiently than UTP. Once incorporated, the analogs inhibit RNA synthesis through immediate chain termination. Omitting the 2'-β-modification alleviates the inhibition of RNA synthesis, highlighting its role in eliciting an antiviral effect. S282T is a well-characterized mutation in motif B of HCV RdRp that confers resistance to sofosbuvir. We discovered that the analogous substitution in YFV (S603T) improved discrimination against the 2'-α-fluoro,2'-β-modified uridine analogs. Collectively, our findings explain their observed anti-YFV activity and identify a conserved mechanism of resistance. Based on its <i>in vivo</i> efficacy and mechanism of action, the 2'-fluoro,2'-bromouridine prodrug shows potential for future therapeutic strategies against YFV.</p>","PeriodicalId":17,"journal":{"name":"ACS Infectious Diseases","volume":" ","pages":"1528-1538"},"PeriodicalIF":3.8000,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12172052/pdf/","citationCount":"0","resultStr":"{\"title\":\"Mechanism of Inhibition of the Active Triphosphate Form of 2'-α-Fluoro,2'-β-bromouridine against Yellow Fever Virus RNA-Dependent RNA Polymerase.\",\"authors\":\"Calvin Joel Gordon, Simon Maximus Walker, Julia Christine LeCher, Franck Amblard, Raymond Felix Schinazi, Matthias Götte\",\"doi\":\"10.1021/acsinfecdis.5c00086\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Yellow fever virus (YFV) is a deadly mosquito-borne flavivirus that infects approximately 200,000 individuals each year. YFV outbreak and infection are persistent throughout South America and Africa, demonstrating significant epidemic potential. Although an approved and effective vaccine exists, the zoonotic nature of YFV undermines any potential eradication efforts, highlighting the need for effective, direct-acting antivirals. Essential for viral replication, RNA-dependent RNA polymerase (RdRp) is a proven therapeutic target. A prominent example includes sofosbuvir, a 2'-α-fluoro,2'-β-methyluridine prodrug approved for the treatment of hepatitis C virus (HCV), that has demonstrated efficacy against YFV <i>in vitro</i>. A structurally similar 2'-α-fluoro, 2'-β-bromouridine prodrug has exhibited potent anti-YFV activity both <i>in vitro</i> and <i>in vivo</i>. Here, we expressed the full-length nonstructural protein 5 from YFV in insect cells to investigate the active triphosphate form of these prodrugs. Enzyme kinetics indicate that both nucleotide analogs are incorporated less efficiently than UTP. Once incorporated, the analogs inhibit RNA synthesis through immediate chain termination. Omitting the 2'-β-modification alleviates the inhibition of RNA synthesis, highlighting its role in eliciting an antiviral effect. S282T is a well-characterized mutation in motif B of HCV RdRp that confers resistance to sofosbuvir. We discovered that the analogous substitution in YFV (S603T) improved discrimination against the 2'-α-fluoro,2'-β-modified uridine analogs. Collectively, our findings explain their observed anti-YFV activity and identify a conserved mechanism of resistance. 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Mechanism of Inhibition of the Active Triphosphate Form of 2'-α-Fluoro,2'-β-bromouridine against Yellow Fever Virus RNA-Dependent RNA Polymerase.
Yellow fever virus (YFV) is a deadly mosquito-borne flavivirus that infects approximately 200,000 individuals each year. YFV outbreak and infection are persistent throughout South America and Africa, demonstrating significant epidemic potential. Although an approved and effective vaccine exists, the zoonotic nature of YFV undermines any potential eradication efforts, highlighting the need for effective, direct-acting antivirals. Essential for viral replication, RNA-dependent RNA polymerase (RdRp) is a proven therapeutic target. A prominent example includes sofosbuvir, a 2'-α-fluoro,2'-β-methyluridine prodrug approved for the treatment of hepatitis C virus (HCV), that has demonstrated efficacy against YFV in vitro. A structurally similar 2'-α-fluoro, 2'-β-bromouridine prodrug has exhibited potent anti-YFV activity both in vitro and in vivo. Here, we expressed the full-length nonstructural protein 5 from YFV in insect cells to investigate the active triphosphate form of these prodrugs. Enzyme kinetics indicate that both nucleotide analogs are incorporated less efficiently than UTP. Once incorporated, the analogs inhibit RNA synthesis through immediate chain termination. Omitting the 2'-β-modification alleviates the inhibition of RNA synthesis, highlighting its role in eliciting an antiviral effect. S282T is a well-characterized mutation in motif B of HCV RdRp that confers resistance to sofosbuvir. We discovered that the analogous substitution in YFV (S603T) improved discrimination against the 2'-α-fluoro,2'-β-modified uridine analogs. Collectively, our findings explain their observed anti-YFV activity and identify a conserved mechanism of resistance. Based on its in vivo efficacy and mechanism of action, the 2'-fluoro,2'-bromouridine prodrug shows potential for future therapeutic strategies against YFV.
期刊介绍:
ACS Infectious Diseases will be the first journal to highlight chemistry and its role in this multidisciplinary and collaborative research area. The journal will cover a diverse array of topics including, but not limited to:
* Discovery and development of new antimicrobial agents — identified through target- or phenotypic-based approaches as well as compounds that induce synergy with antimicrobials.
* Characterization and validation of drug target or pathways — use of single target and genome-wide knockdown and knockouts, biochemical studies, structural biology, new technologies to facilitate characterization and prioritization of potential drug targets.
* Mechanism of drug resistance — fundamental research that advances our understanding of resistance; strategies to prevent resistance.
* Mechanisms of action — use of genetic, metabolomic, and activity- and affinity-based protein profiling to elucidate the mechanism of action of clinical and experimental antimicrobial agents.
* Host-pathogen interactions — tools for studying host-pathogen interactions, cellular biochemistry of hosts and pathogens, and molecular interactions of pathogens with host microbiota.
* Small molecule vaccine adjuvants for infectious disease.
* Viral and bacterial biochemistry and molecular biology.