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deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment
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deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment

Davis, Ryan L ; Homer, Vivienne M ; George, Peter M ; Brennan, Stephen O

Human mutation, 2009-02, Vol.30 (2), p.221-227 [Periódico revisado por pares]

Hoboken: Wiley Subscription Services, Inc., A Wiley Company

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Opportunities and challenges for antisense oligonucleotide therapies
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Opportunities and challenges for antisense oligonucleotide therapies

Kuijper, Elsa C. ; Bergsma, Atze J. ; Pijnappel, W.W.M. Pim ; Aartsma‐Rus, Annemieke

Journal of inherited metabolic disease, 2021-01, Vol.44 (1), p.72-87 [Periódico revisado por pares]

Hoboken, USA: John Wiley & Sons, Inc

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3
Antisense Oligonucleotide (AS‐ODN) Technology: Principle, Mechanism and Challenges
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Antisense Oligonucleotide (AS‐ODN) Technology: Principle, Mechanism and Challenges

Gheibi‐Hayat, Seyed Mohammad ; Jamialahmadi, Khadijeh

Biotechnology and applied biochemistry, 2021-10, Vol.68 (5), p.1086-1094 [Periódico revisado por pares]

United States: Wiley Subscription Services, Inc

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4
Branched Antisense and siRNA Co‐Assembled Nanoplatform for Combined Gene Silencing and Tumor Therapy
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Branched Antisense and siRNA Co‐Assembled Nanoplatform for Combined Gene Silencing and Tumor Therapy

Liu, Jianbing ; Lu, Xuehe ; Wu, Tiantian ; Wu, Xiaohui ; Han, Lin ; Ding, Baoquan

Angewandte Chemie (International ed.), 2021-01, Vol.60 (4), p.1853-1860 [Periódico revisado por pares]

Germany: Wiley Subscription Services, Inc

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Carrier‐Free Delivery of Precise Drug–Chemogene Conjugates for Synergistic Treatment of Drug‐Resistant Cancer
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Carrier‐Free Delivery of Precise Drug–Chemogene Conjugates for Synergistic Treatment of Drug‐Resistant Cancer

Zhu, Lijuan ; Guo, Yuanyuan ; Qian, Qiuhui ; Yan, Deyue ; Li, Yuehua ; Zhu, Xinyuan ; Zhang, Chuan

Angewandte Chemie (International ed.), 2020-10, Vol.59 (41), p.17944-17950 [Periódico revisado por pares]

Germany: Wiley Subscription Services, Inc

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6
Self‐Assembled and Size‐Controllable Oligonucleotide Nanospheres for Effective Antisense Gene Delivery through an Endocytosis‐Independent Pathway
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Self‐Assembled and Size‐Controllable Oligonucleotide Nanospheres for Effective Antisense Gene Delivery through an Endocytosis‐Independent Pathway

Zhou, Jie ; Sun, Liqin ; Wang, Liping ; Liu, Yichang ; Li, Jinyu ; Li, Jingying ; Li, Juan ; Yang, Huanghao

Angewandte Chemie (International ed.), 2019-04, Vol.58 (16), p.5236-5240 [Periódico revisado por pares]

Germany: Wiley Subscription Services, Inc

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7
A Multifunctional Peptide‐Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus
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A Multifunctional Peptide‐Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus

Cheng, Yong ; Sun, Chunli ; Liu, Rui ; Yang, Juliang ; Dai, Jun ; Zhai, Tianyou ; Lou, Xiaoding ; Xia, Fan

Angewandte Chemie (International ed.), 2019-04, Vol.58 (15), p.5049-5053 [Periódico revisado por pares]

Germany: Wiley Subscription Services, Inc

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8
Engineered exosomes for co‐delivery of PGM5‐AS1 and oxaliplatin to reverse drug resistance in colon cancer
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Engineered exosomes for co‐delivery of PGM5‐AS1 and oxaliplatin to reverse drug resistance in colon cancer

Hui, Bingqing ; Lu, Chen ; Wang, Jing ; Xu, Yetao ; Yang, Yuchen ; Ji, Hao ; Li, Xiaofei ; Xu, Lingyan ; Wang, Jiawei ; Tang, Weiwei ; Wang, Keming ; Gu, Yanhong

Journal of cellular physiology, 2022-01, Vol.237 (1), p.911-933 [Periódico revisado por pares]

United States: Wiley Subscription Services, Inc

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9
Development of microRNA therapeutics is coming of age
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Development of microRNA therapeutics is coming of age

Rooij, Eva ; Kauppinen, Sakari

EMBO molecular medicine, 2014-07, Vol.6 (7), p.851-864 [Periódico revisado por pares]

England: John Wiley & Sons, Inc

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10
Spinal muscular atrophy: Diagnosis and management in a new therapeutic era
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Spinal muscular atrophy: Diagnosis and management in a new therapeutic era

Arnold, W. David ; Kassar, Darine ; Kissel, John T.

Muscle & nerve, 2015-02, Vol.51 (2), p.157-167 [Periódico revisado por pares]

United States: Blackwell Publishing Ltd

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