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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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12
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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13
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease
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Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

Park, So Hyun ; Lee, Ciaran M ; Dever, Daniel P ; Davis, Timothy H ; Camarena, Joab ; Srifa, Waracharee ; Zhang, Yankai ; Paikari, Alireza ; Chang, Alicia K ; Porteus, Matthew H ; Sheehan, Vivien A ; Bao, Gang

Nucleic acids research, 2019-09, Vol.47 (15), p.7955-7972 [Periódico revisado por pares]

England: Oxford University Press

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14
The Epigenomics of Cancer
The Epigenomics of Cancer
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The Epigenomics of Cancer

Jones, Peter A. ; Baylin, Stephen B.

Cell, 2007-02, Vol.128 (4), p.683-692 [Periódico revisado por pares]

United States: Elsevier Inc

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15
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
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Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Cideciyan, Artur V. ; Sudharsan, Raghavi ; Dufour, Valérie L. ; Massengill, Michael T. ; Iwabe, Simone ; Swider, Malgorzata ; Lisi, Brianna ; Sumaroka, Alexander ; Marinho, Luis Felipe ; Appelbaum, Tatyana ; Rossmiller, Brian ; Hauswirth, William W. ; Jacobson, Samuel G. ; Lewin, Alfred S. ; Aguirre, Gustavo D. ; Beltran, William A.

Proceedings of the National Academy of Sciences - PNAS, 2018-09, Vol.115 (36), p.E8547-E8556 [Periódico revisado por pares]

United States: National Academy of Sciences

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16
Advances in the delivery of RNA therapeutics: from concept to clinical reality
Advances in the delivery of RNA therapeutics: from concept to clinical reality
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Advances in the delivery of RNA therapeutics: from concept to clinical reality

Kaczmarek, James C ; Kowalski, Piotr S ; Anderson, Daniel G

Genome medicine, 2017-06, Vol.9 (1), p.60-60, Article 60 [Periódico revisado por pares]

England: BioMed Central Ltd

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17
Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cells
Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cells
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Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cells

Belling, Jason N. ; Heidenreich, Liv K. ; Tian, Zhenhua ; Mendoza, Alexandra M. ; Chiou, Tzu-Ting ; Gong, Yao ; Chen, Natalie Y. ; Young, Thomas D. ; Wattanatorn, Natcha ; Park, Jae Hyeon ; Scarabelli, Leonardo ; Chiang, Naihao ; Takahashi, Jack ; Young, Stephen G. ; Stieg, Adam Z. ; De Oliveira, Satiro ; Huang, Tony Jun ; Weiss, Paul S. ; Jonas, Steven J.

Proceedings of the National Academy of Sciences - PNAS, 2020-05, Vol.117 (20), p.10976-10982 [Periódico revisado por pares]

United States: National Academy of Sciences

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18
Systemic delivery of factor IX messenger RNA for protein replacement therapy
Systemic delivery of factor IX messenger RNA for protein replacement therapy
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Systemic delivery of factor IX messenger RNA for protein replacement therapy

Ramaswamy, Suvasini ; Tonnu, Nina ; Tachikawa, Kiyoshi ; Limphong, Pattraranee ; Vega, Jerel B. ; Karmali, Priya P. ; Chivukula, Pad ; Verma, Inder M.

Proceedings of the National Academy of Sciences - PNAS, 2017-03, Vol.114 (10), p.E1941-E1950 [Periódico revisado por pares]

United States: National Academy of Sciences

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19
Development of an intein-mediated split-Cas9 system for gene therapy
Development of an intein-mediated split-Cas9 system for gene therapy
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Development of an intein-mediated split-Cas9 system for gene therapy

Truong, Dong-Jiunn Jeffery ; Kühner, Karin ; Kühn, Ralf ; Werfel, Stanislas ; Engelhardt, Stefan ; Wurst, Wolfgang ; Ortiz, Oskar

Nucleic acids research, 2015-07, Vol.43 (13), p.6450-6458 [Periódico revisado por pares]

England: Oxford University Press

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20
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
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rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis

Shen, Weiran ; Liu, Shengjiang ; Ou, Li

Frontiers in immunology, 2022-10, Vol.13, p.1001263 [Periódico revisado por pares]

Switzerland: Frontiers Media S.A

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