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Refinado por: Base de dados/Biblioteca: EBSCO_MEDLINE Complete（医学期刊全文数据库） remover assunto: Gene Therapy remover
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1
The promise and challenge of therapeutic genome editing
The promise and challenge of therapeutic genome editing
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The promise and challenge of therapeutic genome editing

Doudna, Jennifer A

Nature (London), 2020-02, Vol.578 (7794), p.229-236 [Periódico revisado por pares]

England: Nature Publishing Group

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2
In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models
In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models
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In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models

Yin, Chaoran ; Zhang, Ting ; Qu, Xiying ; Zhang, Yonggang ; Putatunda, Raj ; Xiao, Xiao ; Li, Fang ; Xiao, Weidong ; Zhao, Huaqing ; Dai, Shen ; Qin, Xuebin ; Mo, Xianming ; Young, Won-Bin ; Khalili, Kamel ; Hu, Wenhui

Molecular therapy, 2017-05, Vol.25 (5), p.1168-1186 [Periódico revisado por pares]

United States: Elsevier Inc

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3
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
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In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

Moreno, Ana M. ; Fu, Xin ; Zhu, Jie ; Katrekar, Dhruva ; Shih, Yu-Ru V. ; Marlett, John ; Cabotaje, Jessica ; Tat, Jasmine ; Naughton, John ; Lisowski, Leszek ; Varghese, Shyni ; Zhang, Kang ; Mali, Prashant

Molecular therapy, 2018-07, Vol.26 (7), p.1818-1827 [Periódico revisado por pares]

United States: Elsevier Inc

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4
Targeting microRNAs in cancer: rationale, strategies and challenges
Targeting microRNAs in cancer: rationale, strategies and challenges
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Targeting microRNAs in cancer: rationale, strategies and challenges

Garzon, Ramiro ; Croce, Carlo M ; Marcucci, Guido

Nature reviews. Drug discovery, 2010-10, Vol.9 (10), p.775-789 [Periódico revisado por pares]

England: Nature Publishing Group

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5
Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates and humans
Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates and humans
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Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates and humans

Jüttner, Josephine ; Szabo, Arnold ; Gross-Scherf, Brigitte ; Morikawa, Rei K ; Rompani, Santiago B ; Hantz, Peter ; Szikra, Tamas ; Esposti, Federico ; Cowan, Cameron S ; Bharioke, Arjun ; Patino-Alvarez, Claudia P ; Keles, Özkan ; Kusnyerik, Akos ; Azoulay, Thierry ; Hartl, Dominik ; Krebs, Arnaud R ; Schübeler, Dirk ; Hajdu, Rozina I ; Lukats, Akos ; Nemeth, Janos ; Nagy, Zoltan Z ; Wu, Kun-Chao ; Wu, Rong-Han ; Xiang, Lue ; Fang, Xiao-Long ; Jin, Zi-Bing ; Goldblum, David ; Hasler, Pascal W ; Scholl, Hendrik P N ; Krol, Jacek ; Roska, Botond

Nature neuroscience, 2019-08, Vol.22 (8), p.1345-1356 [Periódico revisado por pares]

United States: Nature Publishing Group

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6
Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction
Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction
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Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction

Sweeney, Colin L. ; Zou, Jizhong ; Choi, Uimook ; Merling, Randall K. ; Liu, Alexander ; Bodansky, Aaron ; Burkett, Sandra ; Kim, Jung-Woong ; De Ravin, Suk See ; Malech, Harry L.

Molecular therapy, 2017-02, Vol.25 (2), p.321-330 [Periódico revisado por pares]

United States: Elsevier Inc

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7
Gold nanoparticle-mediated gene delivery induces widespread changes in the expression of innate immunity genes
Gold nanoparticle-mediated gene delivery induces widespread changes in the expression of innate immunity genes
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Gold nanoparticle-mediated gene delivery induces widespread changes in the expression of innate immunity genes

KIM, E.-Y ; SCHULZ, R ; SWANTEK, P ; KUNSTMAN, K ; MALIM, M. H ; WOLINSKY, S. M

Gene therapy, 2012-03, Vol.19 (3), p.347-353 [Periódico revisado por pares]

London: Nature Publishing Group

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8
A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta‐Thalassemia and Sickle Cell Disease
A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta‐Thalassemia and Sickle Cell Disease
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A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta‐Thalassemia and Sickle Cell Disease

Cai, Liuhong ; Bai, Hao ; Mahairaki, Vasiliki ; Gao, Yongxing ; He, Chaoxia ; Wen, Yanfei ; Jin, You‐Chuan ; Wang, You ; Pan, Rachel L. ; Qasba, Armaan ; Ye, Zhaohui ; Cheng, Linzhao

Stem cells translational medicine, 2018-01, Vol.7 (1), p.87-97 [Periódico revisado por pares]

United States: Oxford University Press

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9
Correction of a pathogenic gene mutation in human embryos
Correction of a pathogenic gene mutation in human embryos
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Correction of a pathogenic gene mutation in human embryos

Ma, Hong ; Marti-Gutierrez, Nuria ; Park, Sang-Wook ; Wu, Jun ; Lee, Yeonmi ; Suzuki, Keiichiro ; Koski, Amy ; Ji, Dongmei ; Hayama, Tomonari ; Ahmed, Riffat ; Darby, Hayley ; Van Dyken, Crystal ; Li, Ying ; Kang, Eunju ; Park, A-Reum ; Kim, Daesik ; Kim, Sang-Tae ; Gong, Jianhui ; Gu, Ying ; Xu, Xun ; Battaglia, David ; Krieg, Sacha A ; Lee, David M ; Wu, Diana H ; Wolf, Don P ; Heitner, Stephen B ; Belmonte, Juan Carlos Izpisua ; Amato, Paula ; Kim, Jin-Soo ; Kaul, Sanjiv ; Mitalipov, Shoukhrat

Nature (London), 2017-08, Vol.548 (7668), p.413-419 [Periódico revisado por pares]

England: Nature Publishing Group

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10
Systemic AAV vectors for widespread and targeted gene delivery in rodents
Systemic AAV vectors for widespread and targeted gene delivery in rodents
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Systemic AAV vectors for widespread and targeted gene delivery in rodents

Challis, Rosemary C ; Ravindra Kumar, Sripriya ; Chan, Ken Y ; Challis, Collin ; Beadle, Keith ; Jang, Min J ; Kim, Hyun Min ; Rajendran, Pradeep S ; Tompkins, John D ; Shivkumar, Kalyanam ; Deverman, Benjamin E ; Gradinaru, Viviana

Nature protocols, 2019-02, Vol.14 (2), p.379-414 [Periódico revisado por pares]

England: Nature Publishing Group

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