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In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation
In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation
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In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation

Liao, Hsin-Kai ; Hatanaka, Fumiyuki ; Araoka, Toshikazu ; Reddy, Pradeep ; Wu, Min-Zu ; Sui, Yinghui ; Yamauchi, Takayoshi ; Sakurai, Masahiro ; O’Keefe, David D. ; Núñez-Delicado, Estrella ; Guillen, Pedro ; Campistol, Josep M. ; Wu, Cheng-Jang ; Lu, Li-Fan ; Esteban, Concepcion Rodriguez ; Izpisua Belmonte, Juan Carlos

Cell, 2017-12, Vol.171 (7), p.1495-1507.e15 [Periódico revisado por pares]

United States: Elsevier Inc

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2
Therapeutic in vivo delivery of gene editing agents
Therapeutic in vivo delivery of gene editing agents
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Therapeutic in vivo delivery of gene editing agents

Raguram, Aditya ; Banskota, Samagya ; Liu, David R.

Cell, 2022-07, Vol.185 (15), p.2806-2827 [Periódico revisado por pares]

United States: Elsevier Inc

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3
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
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Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

Long, Chengzu ; Amoasii, Leonela ; Mireault, Alex A. ; McAnally, John R. ; Li, Hui ; Sanchez-Ortiz, Efrain ; Bhattacharyya, Samadrita ; Shelton, John M. ; Bassel-Duby, Rhonda ; Olson, Eric N.

Science (American Association for the Advancement of Science), 2016-01, Vol.351 (6271), p.400-403 [Periódico revisado por pares]

United States: American Association for the Advancement of Science

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4
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

Tabebordbar, Mohammadsharif ; Zhu, Kexian ; Cheng, Jason K. W. ; Chew, Wei Leong ; Widrick, Jeffrey J. ; Yan, Winston X. ; Maesner, Claire ; Wu, Elizabeth Y. ; Xiao, Ru ; Ran, F. Ann ; Le Cong ; Zhang, Feng ; Vandenberghe, Luk H. ; Church, George M. ; Wagers, Amy J.

Science (American Association for the Advancement of Science), 2016-01, Vol.351 (6271), p.407-411 [Periódico revisado por pares]

United States: American Association for the Advancement of Science

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5
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
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Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Biffi, Alessandra ; Montini, Eugenio ; Lorioli, Laura ; Cesani, Martina ; Fumagalli, Francesca ; Plati, Tiziana ; Baldoli, Cristina ; Martino, Sabata ; Calabria, Andrea ; Canale, Sabrina ; Benedicenti, Fabrizio ; Vallanti, Giuliana ; Biasco, Luca ; Leo, Simone ; Kabbara, Nabil ; Zanetti, Gianluigi ; Rizzo, William B. ; Mehta, Nalini A. L. ; Cicalese, Maria Pia ; Casiraghi, Miriam ; Boelens, Jaap J. ; Del Carro, Ubaldo ; Dow, David J. ; Schmidt, Manfred ; Assanelli, Andrea ; Neduva, Victor ; Di Serio, Clelia ; Stupka, Elia ; Gardner, Jason ; von Kalle, Christof ; Bordignon, Claudio ; Ciceri, Fabio ; Rovelli, Attilio ; Roncarolo, Maria Grazia ; Aiuti, Alessandro ; Sessa, Maria ; Naldini, Luigi

Science (American Association for the Advancement of Science), 2013-08, Vol.341 (6148), p.864-864 [Periódico revisado por pares]

United States: American Association for the Advancement of Science

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6
Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA
Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA
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Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA

Long, Chengzu ; McAnally, John R. ; Shelton, John M. ; Mireault, Alex A. ; Bassel-Duby, Rhonda ; Olson, Eric N.

Science (American Association for the Advancement of Science), 2014-09, Vol.345 (6201), p.1184-1188 [Periódico revisado por pares]

United States: American Association for the Advancement of Science

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7
Oncolytic Viro-Immunotherapy: An Emerging Option in the Treatment of Gliomas
Oncolytic Viro-Immunotherapy: An Emerging Option in the Treatment of Gliomas
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Oncolytic Viro-Immunotherapy: An Emerging Option in the Treatment of Gliomas

Zeng, Jiayi ; Li, Xiangxue ; Sander, Max ; Zhang, Haipeng ; Yan, Guangmei ; Lin, Yuan

Frontiers in immunology, 2021-10, Vol.12, p.721830-721830 [Periódico revisado por pares]

Switzerland: Frontiers Media S.A

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8
Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion
Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion
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Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion

Tse, Longping Victor ; Klinc, Kelli A. ; Madigan, Victoria J. ; Rivera, Ruth M. Castellanos ; Wells, Lindsey F. ; Havlik, L. Patrick ; Smith, J. Kennon ; Agbandje-McKenna, Mavis ; Asokan, Aravind

Proceedings of the National Academy of Sciences - PNAS, 2017-06, Vol.114 (24), p.E4812-E4821 [Periódico revisado por pares]

United States: National Academy of Sciences

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9
mRNA therapeutics in cancer immunotherapy
mRNA therapeutics in cancer immunotherapy
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mRNA therapeutics in cancer immunotherapy

Beck, Jan D ; Reidenbach, Daniel ; Salomon, Nadja ; Sahin, Ugur ; Türeci, Özlem ; Vormehr, Mathias ; Kranz, Lena M

Molecular cancer, 2021-04, Vol.20 (1), p.69-69, Article 69 [Periódico revisado por pares]

England: BioMed Central Ltd

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10
Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration
Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration
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Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

Lagier-Tourenne, Clotilde ; Baughn, Michael ; Rigo, Frank ; Sun, Shuying ; Liu, Patrick ; Li, Hai-Ri ; Jiang, Jie ; Watt, Andrew T ; Chun, Seung ; Katz, Melanie ; Qiu, Jinsong ; Sun, Ying ; Ling, Shuo-Chien ; Zhu, Qiang ; Polymenidou, Magdalini ; Drenner, Kevin ; Artates, Jonathan W ; McAlonis-Downes, Melissa ; Markmiller, Sebastian ; Hutt, Kasey R ; Pizzo, Donald P ; Cady, Janet ; Harms, Matthew B ; Baloh, Robert H ; Vandenberg, Scott R ; Yeo, Gene W ; Fu, Xiang-Dong ; Bennett, C Frank ; Cleveland, Don W ; Ravits, John

Proceedings of the National Academy of Sciences - PNAS, 2013-11, Vol.110 (47), p.E4530-E4539 [Periódico revisado por pares]

United States: National Academy of Sciences

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