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Refinado por: Nome da Publicação: Proceedings Of The National Academy Of Sciences - Pnas remover
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Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy
Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy
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Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy

Lim, Kenji Rowel Q. ; Maruyama, Rika ; Echigoya, Yusuke ; Nguyen, Quynh ; Zhang, Aiping ; Khawaja, Hunain ; Chandra, Sreetama Sen ; Jones, Takako ; Jones, Peter ; Chen, Yi-Wen ; Yokota, Toshifumi

Proceedings of the National Academy of Sciences - PNAS, 2020-07, Vol.117 (28), p.16509-16515 [Periódico revisado por pares]

United States: National Academy of Sciences

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2
Targeting oncogenic KRAS with molecular brush-conjugated antisense oligonucleotides
Targeting oncogenic KRAS with molecular brush-conjugated antisense oligonucleotides
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Targeting oncogenic KRAS with molecular brush-conjugated antisense oligonucleotides

Wang, Dali ; Wang, Qiwei ; Wang, Yuyan ; Chen, Peiru ; Lu, Xueguang ; Jia, Fei ; Sun, Yehui ; Sun, Tingyu ; Zhang, Lei ; Che, Fangyuan ; He, Jialu ; Lian, Liming ; Morano, Gemma ; Shen, Michael ; Ren, Mengqi ; Dong, Sijia S ; Zhao, Jean J ; Zhang, Ke

Proceedings of the National Academy of Sciences - PNAS, 2022-07, Vol.119 (29), p.e2113180119 [Periódico revisado por pares]

United States: National Academy of Sciences

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3
Exon-skipping antisense oligonucleotides for cystic fibrosis therapy
Exon-skipping antisense oligonucleotides for cystic fibrosis therapy
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Exon-skipping antisense oligonucleotides for cystic fibrosis therapy

Kim, Young Jin ; Sivetz, Nicole ; Layne, Jessica ; Voss, Dillon M ; Yang, Lucia ; Zhang, Qian ; Krainer, Adrian R

Proceedings of the National Academy of Sciences - PNAS, 2022-01, Vol.119 (3) [Periódico revisado por pares]

United States: National Academy of Sciences

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4
Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency
Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency
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Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency

Wood, Mattew J. A. ; Altman, Sidney

Proceedings of the National Academy of Sciences - PNAS, 2020-12, Vol.117 (51), p.32370-32379 [Periódico revisado por pares]

United States: National Academy of Sciences

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5
A systems approach delivers a functional microRNA catalog and expanded targets for seizure suppression in temporal lobe epilepsy
A systems approach delivers a functional microRNA catalog and expanded targets for seizure suppression in temporal lobe epilepsy
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A systems approach delivers a functional microRNA catalog and expanded targets for seizure suppression in temporal lobe epilepsy

Venø, Morten T. ; Reschke, Cristina R. ; Morris, Gareth ; Connolly, Niamh M. C. ; Su, Junyi ; Yan, Yan ; Engel, Tobias ; Jimenez-Mateos, Eva M. ; Harder, Lea M. ; Pultz, Dennis ; Haunsberger, Stefan J. ; Pal, Ajay ; Heller, Janosch P. ; Campbell, Aoife ; Langa, Elena ; Brennan, Gary P. ; Conboy, Karen ; Richardson, Amy ; Norwood, Braxton A. ; Costard, Lara S. ; Neubert, Valentin ; Del Gallo, Federico ; Salvettik, Beatrice ; Vangoor, Vamshidhar R. ; Sanz-Rodriguez, Amaya ; Muilu, Juha ; Fabene, Paolo F. ; Pasterkamp, R. Jeroen ; Prehn, Jochen H. M. ; Schorge, Stephanie ; Andersen, Jens S. ; Rosenow, Felix ; Bauer, Sebastian ; Kjems, Jørgen ; Henshallb, David C.

Proceedings of the National Academy of Sciences - PNAS, 2020-07, Vol.117 (27), p.15977-15988 [Periódico revisado por pares]

United States: National Academy of Sciences

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6
A bipartite boundary element restricts UBE3A imprinting to mature neurons
A bipartite boundary element restricts UBE3A imprinting to mature neurons
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A bipartite boundary element restricts UBE3A imprinting to mature neurons

Hsiao, Jack S. ; Germain, Noelle D. ; Wilderman, Andrea ; Stoddard, Christopher ; Wojenski, Luke A. ; Villafano, Geno J. ; Core, Leighton ; Cotney, Justin ; Chamberlain, Stormy J.

Proceedings of the National Academy of Sciences - PNAS, 2019-02, Vol.116 (6), p.2181-2186 [Periódico revisado por pares]

United States: National Academy of Sciences

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7
Antisense oligonucleotide rescue of CGG expansion-dependent FMR1 mis-splicing in fragile X syndrome restores FMRP
Antisense oligonucleotide rescue of CGG expansion-dependent FMR1 mis-splicing in fragile X syndrome restores FMRP
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Antisense oligonucleotide rescue of CGG expansion-dependent FMR1 mis-splicing in fragile X syndrome restores FMRP

Shah, Sneha ; Sharp, Kevin J ; Raju Ponny, Sithara ; Lee, Jonathan ; Watts, Jonathan K ; Berry-Kravis, Elizabeth ; Richter, Joel D

Proceedings of the National Academy of Sciences - PNAS, 2023-07, Vol.120 (27), p.e2302534120-e2302534120 [Periódico revisado por pares]

United States: National Academy of Sciences

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8
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

McBride, Jodi L ; Boudreau, Ryan L ; Harper, Scott Q ; Staber, Patrick D ; Monteys, Alex Mas ; Martins, Inâs ; Gilmore, Brian L ; Burstein, Haim ; Peluso, Richard W ; Polisky, Barry ; Carter, Barrie J ; Davidson, Beverly L

Proceedings of the National Academy of Sciences - PNAS, 2008-04, Vol.105 (15), p.5868-5873 [Periódico revisado por pares]

United States: National Academy of Sciences

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9
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
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Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

Wu, Bo ; Moulton, Hong M ; Iversen, Patrick L ; Jiang, Jiangang ; Li, Juan ; Li, Jianbin ; Spurney, Christopher F ; Sali, Arpana ; Guerron, Alfredo D ; Nagaraju, Kanneboyina ; Doran, Timothy ; Lu, Peijuan ; Xiao, Xiao ; Lu, Qi Long

Proceedings of the National Academy of Sciences - PNAS, 2008-09, Vol.105 (39), p.14814-14819 [Periódico revisado por pares]

United States: National Academy of Sciences

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10
Topical delivery of siRNA-based spherical nucleic acid nanoparticle conjugates for gene regulation
Topical delivery of siRNA-based spherical nucleic acid nanoparticle conjugates for gene regulation
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Topical delivery of siRNA-based spherical nucleic acid nanoparticle conjugates for gene regulation

Zheng, Dan ; Giljohann, David A ; Chen, David L ; Massich, Matthew D ; Wang, Xiao-Qi ; Iordanov, Hristo ; Mirkin, Chad A ; Paller, Amy S

Proceedings of the National Academy of Sciences - PNAS, 2012-07, Vol.109 (30), p.11975-11980 [Periódico revisado por pares]

United States: National Academy of Sciences

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