MicroRNA‐dependent regulation of DNA methyltransferase‐1 and tumor suppressor gene expression by interleukin‐6 in human malignant cholangiocytes von Braconi, Chiara, Huang, Nianyuan, Patel, Tushar

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Endothelial nitric oxide signaling regulates Notch1 in aortic valve disease von Bosse, Kevin, Hans, Chetan P, Zhao, Ning, Koenig, Sara N, Huang, Nianyuan, Guggilam, Anuradha, LaHaye, Stephanie, Tao, Ge, Lucchesi, Pamela A, Lincoln, Joy, Lilly, Brenda, Garg, Vidu

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Expression and functional role of a transcribed noncoding RNA with an ultraconserved element in hepatocellular carcinoma von Braconi, Chiara, Valeri, Nicola, Kogure, Takayuki, Gasparini, Pierluigi, Huang, Nianyuan, Nuovo, Gerard J., Terracciano, Luigi, Croce, Carlo M., Patel, Tushar, White, Raymond L.

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Hepatitis C Virus Proteins Modulate MicroRNA Expression and Chemosensitivity in Malignant Hepatocytes von BRACONI, Chiara, VALERI, Nicola, GASPARINI, Pierluigi, NIANYUAN HUANG, TACCIOLI, Cristian, NUOVO, Gerard, SUZUKI, Tetsuro, CROCE, Carlo Maria, PATEL, Tushar

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Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping von Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.

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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse von Wein, Nicolas, Vetter, Tatyana A., Vulin, Adeline, Simmons, Tabatha R., Frair, Emma C., Bradley, Adrienne J., Gushchina, Liubov V., Almeida, Camila F., Huang, Nianyuan, Lesman, Daniel, Rajakumar, Dhanarajan, Weiss, Robert B., Flanigan, Kevin M.

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Targeting the IL-6 dependent phenotype can identify novel therapies for cholangiocarcinoma von Braconi, Chiara, Swenson, Erica, Kogure, Takayuki, Huang, Nianyuan, Patel, Tushar

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Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy von Gushchina, Liubov V., Vetter, Tatyana A., Frair, Emma C., Bradley, Adrienne J., Grounds, Kelly M., Lay, Jacob W., Huang, Nianyuan, Suhaiba, Aisha, Schnell, Frederick J., Hanson, Gunnar, Simmons, Tabatha R., Wein, Nicolas, Flanigan, Kevin M.

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Candidate therapeutic agents for hepatocellular cancer can be identified from phenotype‐associated gene expression signatures von Braconi, Chiara, Meng, Fanyin, Swenson, Erica, Khrapenko, Lyudmyla, Huang, Nianyuan, Patel, Tushar

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Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines von Almeida, Camila F, Robriquet, Florence, Vetter, Tatyana A, Huang, Nianyuan, Neinast, Reid, Hernandez-Rosario, Lumariz, Rajakumar, Dhanarajan, Arnold, W David, McBride, Kim L, Flanigan, Kevin M, Weiss, Robert B, Wein, Nicolas

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Disruption of myocardial Gata4 and Tbx5 results in defects in cardiomyocyte proliferation and atrioventricular septation von Misra, Chaitali, Chang, Sheng-Wei, Basu, Madhumita, Huang, Nianyuan, Garg, Vidu

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Inhibition of Notch1 Signaling Reduces Abdominal Aortic Aneurysm in Mice by Attenuating Macrophage-Mediated Inflammation von Hans, Chetan P, Koenig, Sara N, Huang, Nianyuan, Cheng, Jeeyun, Beceiro, Susana, Guggilam, Anuradha, Kuivaniemi, Helena, Partida-Sánchez, Santiago, Garg, Vidu

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624. A Single Neonatal Delivery of an Exon 2 Directed AAV9.U7snRNA Vector Results in Long-Term Dystrophin Expression That Prevents Pathologic Features in the Dup2 Mouse von Wein, Nicolas, Simmons, Tabatha R., Gumienny, Felecia, Yurkoski, Jacqueline, Huang, Nianyuan, Muntoni, Francesco, Flanigan, Kevin M.

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Genetic Abnormalities in FOXP1 Are Associated with Congenital Heart Defects von Chang, Sheng-Wei, Mislankar, Mona, Misra, Chaitali, Huang, Nianyuan, DaJusta, Daniel G., Harrison, Steven M., McBride, Kim L., Baker, Linda A., Garg, Vidu

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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders von Almeida, Camila F., Frair, Emma C., Huang, Nianyuan, Neinast, Reid, McBride, Kim L., Weiss, Robert B., Flanigan, Kevin M., Wein, Nicolas

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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders von Almeida, Camila F, Frair, Emma C, Huang, Nianyuan, Neinast, Reid, McBride, Kim L, Weiss, Robert B, Flanigan, Kevin M, Wein, Nicolas

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60. Intramuscular and Systemic Induction of the N-Truncated Dystrophin By Out-Of-Frame Exon 2 Skipping Restores Muscle Function in the Dup2 Mouse, Providing Further Support for a T... von Wein, Nicolas, Vulin, Adeline, Simmons, Tabatha, Gumienny, Felecia, Huang, Nianyuan, Muntoni, Francesco, Ervasti, James, Weiss, Robert, Flanigan, Kevin

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MicroRNA dependent regulation of DNMT-1 and tumor suppressor gene expression by Interleukin-6 in human malignant cholangiocytes von Braconi, Chiara, Huang, Nianyuan, Patel, Tushar

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Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach von Wein, Nicolas, Vulin, Adeline, Findlay, Andrew R., Gumienny, Felecia, Huang, Nianyuan, Wilton, Steve D., Flanigan, Kevin M.

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Inhibition of Notchl Signaling Reduces Abdominal Aortic Aneurysm in Mice by Attenuating Macrophage-Mediated Inflammation von HANS, Chetan P, KOENIG, Sara N, NIANYUAN HUANG, CHENG, Jeeyun, BECEIRO, Susana, GUGGILAM, Anuradha, KUIVANIEMI, Helena, PARTIDA-SANCHEZ, Santiago, GARG, Vidu

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