Molecular TherapyPub Date : 2025-01-09DOI: 10.1016/j.ymthe.2025.01.008
Maureen Buckley, Mariluz Araínga, Laura Maiorino, Ivan S Pires, B J Kim, Katarzyna Kaczmarek Michaels, Jonathan Dye, Kashif Qureshi, Yiming Zhang, Howard Mak, Jon M Steichen, William R Schief, Francois Villinger, Darrell J Irvine
{"title":"Visualizing lipid nanoparticle trafficking for mRNA vaccine delivery in non-human primates.","authors":"Maureen Buckley, Mariluz Araínga, Laura Maiorino, Ivan S Pires, B J Kim, Katarzyna Kaczmarek Michaels, Jonathan Dye, Kashif Qureshi, Yiming Zhang, Howard Mak, Jon M Steichen, William R Schief, Francois Villinger, Darrell J Irvine","doi":"10.1016/j.ymthe.2025.01.008","DOIUrl":"10.1016/j.ymthe.2025.01.008","url":null,"abstract":"<p><p>mRNA delivered using lipid nanoparticles (LNPs) has become an important subunit vaccine modality, but mechanisms of action for mRNA vaccines remain incompletely understood. Here, we synthesized a metal chelator-lipid conjugate enabling positron emission tomography (PET) tracer labeling of LNP/mRNA vaccines for quantitative visualization of vaccine trafficking in live mice and non-human primates (NHPs). Following i.m. injection, we observed LNPs distributing through injected muscle tissue, simultaneous with rapid trafficking to draining lymph nodes (dLNs). Deltoid injection of LNPs mimicking human vaccine administration led to stochastic LNP delivery to 3 different sets of dLNs. LNP uptake in dLNs was confirmed by histology, and cellular analysis of tissues via flow cytometry identified antigen-presenting cells as the primary cell type responsible for early LNP uptake and mRNA translation. These results provide insights into the biodistribution of mRNA vaccines administered at clinically relevant doses, injection volumes, and injection sites in an important large animal model for vaccine development.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":""},"PeriodicalIF":12.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-09DOI: 10.1016/j.ymthe.2025.01.011
Irene Carrozzo, Giulia Maule, Carmelo Gentile, Alessandro Umbach, Matteo Ciciani, Daniela Guidone, Martina De Santis, Gianluca Petris, Luis Juan Vicente Galietta, Daniele Arosio, Anna Cereseto
{"title":"Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing.","authors":"Irene Carrozzo, Giulia Maule, Carmelo Gentile, Alessandro Umbach, Matteo Ciciani, Daniela Guidone, Martina De Santis, Gianluca Petris, Luis Juan Vicente Galietta, Daniele Arosio, Anna Cereseto","doi":"10.1016/j.ymthe.2025.01.011","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.01.011","url":null,"abstract":"<p><p>Cystic Fibrosis (CF) is a life-shortening autosomal recessive disease caused by mutations in the CFTR gene, resulting in functional impairment of the encoded ion channel. F508del mutation, a trinucleotide deletion, is the most frequent cause of CF affecting approximately 80% of persons with cystic fibrosis (pwCFs). Even though current pharmacological treatments alleviate the F508del-CF disease symptoms there is no definitive cure. Here we leveraged revertant mutations (RMs) in cis with F508del to rescue CFTR protein folding and restore its function. We developed CRISPR base editing strategies to efficiently and precisely introduce the desired mutations in the F508del locus. Both editing and CFTR function recovery were verified in CF cellular models including primary epithelial cells derived from pwCFs. The efficacy of the CFTR recovery strategy was validated in cultures of pseudostratified epithelia from pwCF cells showing full recovery of ion transport. Additionally, we observed an additive effect by combining our strategy with small molecules that enhance F508del activity, thus paving the way to combinatorial therapies.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":""},"PeriodicalIF":12.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966028","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-09DOI: 10.1016/j.ymthe.2025.01.005
Narae Kim, Yohei Yokobayashi
{"title":"Scalable control of stem cell fate by riboswitch-regulated RNA viral vector without genomic integration.","authors":"Narae Kim, Yohei Yokobayashi","doi":"10.1016/j.ymthe.2025.01.005","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.01.005","url":null,"abstract":"<p><p>Transgene expression in stem cells is a powerful means of regulating cellular properties and differentiation into various cell types. However, existing vectors for transgene expression in stem cells suffer from limitations such as the need for genomic integration, the transient nature of gene expression, and the inability to temporally regulate transgene expression, which hinder biomedical and clinical applications. Here we report a new class of RNA virus-based vectors for scalable and integration-free transgene expression in mouse embryonic stem cells (mESCs). The vector is equipped with a small molecule-regulated riboswitch and a drug selection marker that allow temporal regulation of transgene expression and stable maintenance of the vector in proliferating stem cells. We demonstrated the utility of the vector by maintaining the pluripotency of mESCs in a differentiation induction medium by expressing Nanog and inducing myogenic differentiation by triggering Myod1 expression, without altering the mESC genome.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":""},"PeriodicalIF":12.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966029","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Focused Ultrasound and Microbubble-Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein to Human Induced Pluripotent Stem Cells.","authors":"Kyle Hazel, Davindra Singh, Stephanie He, Zakary Guertin, Mathieu C Husser, Brandon Helfield","doi":"10.1016/j.ymthe.2025.01.013","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.01.013","url":null,"abstract":"<p><p>CRISPR-Cas9 ribonucleoproteins (RNPs) have been heavily considered for gene therapy due to their high on-target efficiency, rapid activity and lack of insertional mutagenesis relative to other CRISPR-Cas9 delivery formats. Genetic diseases such as hypertrophic cardiomyopathy currently lack effective treatment strategies and are prime targets for CRISPR-Cas9 gene editing technology. However, current in-vivo delivery strategies for Cas9 pose risks of unwanted immunogenic responses. This proof-of-concept study aimed to demonstrate that focused ultrasound (FUS) in combination with microbubbles can be used to deliver Cas9-sgRNA (single guide RNA) RNPs and functionally edit human induced pluripotent stem cells (hiPSCs) in-vitro, a model system that can be expanded to cardiovascular research via hiPSC-derived cardiomyocytes. Here, we first determine acoustic conditions suitable for the viable delivery of large proteins to hiPSC with clinical Definity® microbubble agents using our customized experimental platform. From here, we delivered Cas9-sgRNA RNP complexes targeting the EGFP (enhanced green fluorescent protein) gene to EGFP-expressing hiPSCs for EGFP knockout. Simultaneous acoustic cavitation detection during treatment confirmed a strong correlation between microbubble disruption and viable FUS-mediated protein delivery in hiPSCs. This study shows, for the first time, the potential for an FUS-mediated technique for targeted and precise CRISPR-Cas9 gene editing in human stem cells.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":""},"PeriodicalIF":12.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966000","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-09DOI: 10.1016/j.ymthe.2025.01.012
Guocheng Zhong, Xiaomin Zhang, Ruocong Zhao, Zheng Guo, Chenguang Wang, Chuan Yu, Dongzhe Liu, Ke Hu, Yujie Gao, Bochen Zhao, Xianhao Liu, Xuanren Shi, Lei Chen, Yisheng Li, Li Yu
{"title":"The high efficacy of claudin18.2-targeted CAR-T cell therapy in advanced pancreatic cancer with a strategy to ensure the safety of patients.","authors":"Guocheng Zhong, Xiaomin Zhang, Ruocong Zhao, Zheng Guo, Chenguang Wang, Chuan Yu, Dongzhe Liu, Ke Hu, Yujie Gao, Bochen Zhao, Xianhao Liu, Xuanren Shi, Lei Chen, Yisheng Li, Li Yu","doi":"10.1016/j.ymthe.2025.01.012","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.01.012","url":null,"abstract":"<p><p>Pancreatic cancer (PC) is one of the most lethal digestive system tumors. Claudin18.2 is highly expressed in PC tissue and could serve as a suitable target for CAR-T therapy. In the present study, we reported the utilization of tEGFR-expressing claudin18.2-targeted CAR-T cells to treat 3 patients with advanced PC. Intriguingly, all 3 patients achieved disease remission after CAR-T cell infusion, with 1 complete remission (CR) and 2 partial remissions (PR). However, gastric mucosal injury was observed, which was recognized as on-target off-tumor toxicity (OTOT) and may be due to the expression of claudin18.2 on normal gastric tissues. To control the severe OTOT in patient 3, cyclophosphamide and cetuximab were administered to deplete CAR-T cells, and they successfully controlled OTOT. Single cell transcriptome and TCR sequencing revealed the objective alterations of CAR-T cell clones after cetuximab treatment. Collectively, the present study showed the robust anti-tumor activity of claudin18.2-targeted CAR-T cells against PC, and reported the feasibility of antibody-dependent safety switch strategy to control the OTOT caused by CAR-T cells in patients. Our study may pave the way for the development of a novel strategy to treat patients with advanced PC in the future.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":""},"PeriodicalIF":12.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966030","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-08Epub Date: 2024-11-19DOI: 10.1016/j.ymthe.2024.11.029
Zachary C E Hawley, Ingrid D Pardo, Shaolong Cao, Maria I Zavodszky, Fergal Casey, Kyle Ferber, Yi Luo, Sam Hana, Shukkwan K Chen, Jessica Doherty, Raquel Costa, Patrick Cullen, Yuqing Liu, Thomas M Carlile, Twinkle Chowdhury, Benjamin Doyle, Pete Clarner, Kevin Mangaudis, Edward Guilmette, Shawn Bourque, David Koske, Murali V P Nadella, Patrick Trapa, Michael L Hawes, Denitza Raitcheva, Shih-Ching Lo
{"title":"Dorsal root ganglion toxicity after AAV intra-CSF delivery of a RNAi expression construct into non-human primates and mice.","authors":"Zachary C E Hawley, Ingrid D Pardo, Shaolong Cao, Maria I Zavodszky, Fergal Casey, Kyle Ferber, Yi Luo, Sam Hana, Shukkwan K Chen, Jessica Doherty, Raquel Costa, Patrick Cullen, Yuqing Liu, Thomas M Carlile, Twinkle Chowdhury, Benjamin Doyle, Pete Clarner, Kevin Mangaudis, Edward Guilmette, Shawn Bourque, David Koske, Murali V P Nadella, Patrick Trapa, Michael L Hawes, Denitza Raitcheva, Shih-Ching Lo","doi":"10.1016/j.ymthe.2024.11.029","DOIUrl":"10.1016/j.ymthe.2024.11.029","url":null,"abstract":"<p><p>Dorsal root ganglion (DRG) toxicity has been consistently reported as a potential safety concern after delivery of adeno-associated viruses (AAVs) containing gene-replacement vectors but has yet to be reported for RNAi-based vectors. Here, we report DRG toxicity after AAV intra-CSF delivery of an RNAi expression construct-artificial microRNA targeting superoxide dismutase 1 (SOD1)-in non-human primates (NHPs) and provide evidence that this can be recapitulated within mice. Histopathology evaluation showed that NHPs and mice develop DRG toxicity after AAV delivery, including DRG neuron degeneration and necrosis and nerve-fiber degeneration that were associated with increases in cerebrospinal fluid (CSF) and serum phosphorylated neurofilament heavy chain (pNF-H). RNA-sequencing analysis of DRGs showed that dysregulated pathways were preserved between NHPs and mice, including increases in innate/adaptive immune responses and decreases in mitochondrial- and neuronal-related genes, following AAV treatment. Finally, endogenous miR-21-5p was upregulated in DRGs of AAV-treated NHPs and mice. Increases in miR-21-5p were also identified within the CSF of NHPs, which significantly correlated with pNF-H, implicating miR-21-5p as a potential biomarker of DRG toxicity in conjunction with other molecular analytes. This work highlights the importance of assessing safety concerns related to DRG toxicity when developing RNAi-based AAV vectors for therapeutic purposes.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"215-234"},"PeriodicalIF":12.1,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142676021","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-08Epub Date: 2024-11-06DOI: 10.1016/j.ymthe.2024.11.004
Anastasiya Kostyusheva, Sergey Brezgin, Natalia Ponomareva, Anastasiia Frolova, Alexander Lunin, Ekaterina Bayurova, Andrey Tikhonov, Olga Slatinskaya, Polina Demina, Artyom Kachanov, Gulalek Babayeva, Irina Khan, Dmitry Khochenkov, Yulia Khochenkova, Darina Sokolova, Denis Silachev, Georgy Maksimov, Evgeny Khaydukov, Vadim S Pokrovsky, Andrey A Zamyatnin, Alessandro Parodi, Ilya Gordeychuk, Vladimir Chulanov, Dmitry Kostyushev
{"title":"Biologics-based technologies for highly efficient and targeted RNA delivery.","authors":"Anastasiya Kostyusheva, Sergey Brezgin, Natalia Ponomareva, Anastasiia Frolova, Alexander Lunin, Ekaterina Bayurova, Andrey Tikhonov, Olga Slatinskaya, Polina Demina, Artyom Kachanov, Gulalek Babayeva, Irina Khan, Dmitry Khochenkov, Yulia Khochenkova, Darina Sokolova, Denis Silachev, Georgy Maksimov, Evgeny Khaydukov, Vadim S Pokrovsky, Andrey A Zamyatnin, Alessandro Parodi, Ilya Gordeychuk, Vladimir Chulanov, Dmitry Kostyushev","doi":"10.1016/j.ymthe.2024.11.004","DOIUrl":"10.1016/j.ymthe.2024.11.004","url":null,"abstract":"<p><p>The demand for RNA-based therapeutics is increasing globally. However, their use is hampered by the lack of safe and effective delivery vehicles. Here, we developed technologies for highly efficient delivery of RNA cargo into programmable extracellular vesicle-mimetic nanovesicles (EMNVs) by fabricating hybrid EMNV-liposomes (Hybs). Tissue targeting is endowed by highly efficient genetic platforms based on truncated CD63 (ΔCD63) or PTGFRN proteins. For the first time we reveal their efficiency in functionalizing EMNVs, resulting in >10-fold enhancement of nanoparticle internalization in vitro and >2-fold in vivo. RNA delivery using Hybs demonstrated efficiency of >85% in human and mouse cell lines. Comparative analysis of EMNVs and Hyb lysosome colocalization and stability suggested that Hybs enter the lysosomal compartment and escape over time, whereas EMNVs primarily avoid it. Finally, we used these technologies to generate liver-targeting Hybs loaded with therapeutic small interfering RNA and demonstrated the robust efficiency of this system in vitro and in vivo. These technologies can be adapted for manufacturing a wide range of next-generation vehicles for highly efficient, safe delivery of RNA into desired organs and tissues for therapeutic and prophylactic applications.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"168-183"},"PeriodicalIF":12.1,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142604742","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-08Epub Date: 2024-10-09DOI: 10.1016/j.ymthe.2024.10.003
Yanhong Jiang, Shuanghong Chen, Shenlin Hsiao, Haokun Zhang, Da Xie, Zi Jun Wang, Wendan Ren, Mingyao Liu, Jiaoyang Liao, Yuxuan Wu
{"title":"Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption.","authors":"Yanhong Jiang, Shuanghong Chen, Shenlin Hsiao, Haokun Zhang, Da Xie, Zi Jun Wang, Wendan Ren, Mingyao Liu, Jiaoyang Liao, Yuxuan Wu","doi":"10.1016/j.ymthe.2024.10.003","DOIUrl":"10.1016/j.ymthe.2024.10.003","url":null,"abstract":"<p><p>Primary hyperoxaluria type 1 (PH1) is a severe genetic metabolic disorder caused by mutations in the AGXT gene, leading to defects in enzymes crucial for glyoxylate metabolism. PH1 is characterized by severe, potentially life-threatening manifestations due to excessive oxalate accumulation, which leads to calcium oxalate crystal deposits in the kidneys and, ultimately, renal failure and systemic oxalosis. Existing substrate reduction therapies, such as inhibition of liver-specific glycolate oxidase (GO) encoded by HAO1 using siRNA or CRISPR-Cas9 delivered by adeno-associated virus, either require repeated dosing or have raised safety concerns. To address these limitations, our study employed lipid nanoparticles (LNPs) for CRISPR-Cas9 delivery to rapidly generate a PH1 mouse model and validate the therapeutic efficacy of LNP-CRISPR-Cas9 targeting the Hao1 gene. The LNP-CRISPR-Cas9 system exhibited efficient editing of the Hao1 gene, significantly reducing GO expression and lowering urinary oxalate levels in treated PH1 mice. Notably, these effects persisted for 12 months with no significant off-target effects, liver-induced toxicity, or substantial immune responses, highlighting the approach's safety and specificity. Furthermore, the developed humanized mouse model validated the efficacy of our therapeutic strategy. These findings support LNP-CRISPR-Cas9 targeting HAO1 as a promising and safer alternative for PH1 treatment with a single administration.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"104-118"},"PeriodicalIF":12.1,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142391979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"C-reactive protein promotes diabetic kidney disease via Smad3-mediated NLRP3 inflammasome activation.","authors":"Yifan Wang, Yong-Ke You, Jianbo Guo, Jianan Wang, Baoyi Shao, Haidi Li, Xiaoming Meng, Hui-Yao Lan, Haiyong Chen","doi":"10.1016/j.ymthe.2024.11.018","DOIUrl":"10.1016/j.ymthe.2024.11.018","url":null,"abstract":"<p><p>Diabetic kidney disease (DKD) is the leading cause of end-stage kidney diseases resulting in enormous socio-economic burden. Accumulated evidence has indicated that C-reactive protein (CRP) exacerbates DKD by enhancing renal inflammation and fibrosis through TGF-β/Smad3 signaling. NLRP3 inflammasome is the key sensor contributing to renal inflammation. However, whether CRP enhances inflammation in DKD via NLRP3 inflammasome-related pathway remains unknown. In this study, we demonstrate that CRP promotes DKD via Smad3-mediated NLRP3 inflammasome activation as mice overexpressing human CRP gene exhibits accelerated renal inflammation in diabetic kidneys, which is associated with the activation of Smad3 and NLRP3 inflammasomes. In contrast, blockade of CPR signaling with a neutralizing anti-CD32 antibody attenuates CRP-induced activation of Smad3 and NLRP3 in vitro. Importantly, genetic deletion or pharmacological inhibition of Smad3 also mitigates CRP-induced activation of NLRP3 in diabetic kidneys or in high glucose-treated cells. Mechanistically, we reveal that Smad3 binds to the NLRP3 gene promoter, which is enhanced by CRP. Taken together, we conclude that CRP induces renal inflammation in DKD via a Smad3-NLRP3 inflammasome-dependent mechanism. Thus, targeting CRP or Smad3-NLRP3 pathways may be a new therapeutic potential for DKD.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"263-278"},"PeriodicalIF":12.1,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142624234","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-01-08Epub Date: 2024-12-06DOI: 10.1016/j.ymthe.2024.12.002
William W Du, Weining Yang, Xiangmin Li, Ling Fang, Nan Wu, Feiya Li, Yu Chen, Qihan He, Elizabeth Liu, Zhenguo Yang, Faryal Mehwish Awan, Mingyao Liu, Burton B Yang
{"title":"The Circular RNA circSKA3 Binds Integrin β1 to Induce Invadopodium Formation Enhancing Breast Cancer Invasion.","authors":"William W Du, Weining Yang, Xiangmin Li, Ling Fang, Nan Wu, Feiya Li, Yu Chen, Qihan He, Elizabeth Liu, Zhenguo Yang, Faryal Mehwish Awan, Mingyao Liu, Burton B Yang","doi":"10.1016/j.ymthe.2024.12.002","DOIUrl":"https://doi.org/10.1016/j.ymthe.2024.12.002","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"33 1","pages":"416"},"PeriodicalIF":12.1,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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