Pesquisa | Secretaria de Estado da Saúde

1.

Past, present, and future of CRISPR genome editing technologies.

Pacesa, Martin; Pelea, Oana; Jinek, Martin.

Cell ; 187(5): 1076-1100, 2024 Feb 29.

Artigo Inglês | MEDLINE | ID: mdl-38428389

2.

Muscle fusogens go viral for gene delivery to skeletal muscle.

Gibbs, Devin E; Pyle, April D.

Cell ; 186(10): 2041-2043, 2023 05 11.

Artigo Inglês | MEDLINE | ID: mdl-37172560

3.

An AsCas12f-based compact genome-editing tool derived by deep mutational scanning and structural analysis.

Hino, Tomohiro; Omura, Satoshi N; Nakagawa, Ryoya; Togashi, Tomoki; Takeda, Satoru N; Hiramoto, Takafumi; Tasaka, Satoshi; Hirano, Hisato; Tokuyama, Takeshi; Uosaki, Hideki; Ishiguro, Soh; Kagieva, Madina; Yamano, Hiroyuki; Ozaki, Yuki; Motooka, Daisuke; Mori, Hideto; Kirita, Yuhei; Kise, Yoshiaki; Itoh, Yuzuru; Matoba, Satoaki; Aburatani, Hiroyuki; Yachie, Nozomu; Karvelis, Tautvydas; Siksnys, Virginijus; Ohmori, Tsukasa; Hoshino, Atsushi; Nureki, Osamu.

Cell ; 186(22): 4920-4935.e23, 2023 10 26.

Artigo Inglês | MEDLINE | ID: mdl-37776859

4.

Adenoviral gene therapy for bladder cancer.

Herzog, Roland W; Suzuki, Masataka.

Cell ; 186(5): 893, 2023 03 02.

Artigo Inglês | MEDLINE | ID: mdl-36868210

5.

Herpes simplex virus gene therapy for dystrophic epidermolysis bullosa (DEB).

Epstein, Alberto L; Haag-Molkenteller, Cornelia.

Cell ; 186(17): 3523-3523.e1, 2023 08 17.

Artigo Inglês | MEDLINE | ID: mdl-37595560

6.

Gene therapy for inborn errors of immunity: Base editing comes into play.

Malech, Harry L; Notarangelo, Luigi D.

Cell ; 186(7): 1302-1304, 2023 03 30.

Artigo Inglês | MEDLINE | ID: mdl-37001495

7.

Circuit-specific gene therapy reverses core symptoms in a primate Parkinson's disease model.

Chen, Yefei; Hong, Zexuan; Wang, Jingyi; Liu, Kunlin; Liu, Jing; Lin, Jianbang; Feng, Shijing; Zhang, Tianhui; Shan, Liang; Liu, Taian; Guo, Pinyue; Lin, Yunping; Li, Tian; Chen, Qian; Jiang, Xiaodan; Li, Anan; Li, Xiang; Li, Yuantao; Wilde, Jonathan J; Bao, Jin; Dai, Ji; Lu, Zhonghua.

Cell ; 186(24): 5394-5410.e18, 2023 11 22.

Artigo Inglês | MEDLINE | ID: mdl-37922901

8.

Therapeutic in vivo delivery of gene editing agents.

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

Cell ; 185(15): 2806-2827, 2022 07 21.

Artigo Inglês | MEDLINE | ID: mdl-35798006

9.

Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells.

Omer-Javed, Attya; Pedrazzani, Gabriele; Albano, Luisa; Ghaus, Sherash; Latroche, Claire; Manzi, Maura; Ferrari, Samuele; Fiumara, Martina; Jacob, Aurelien; Vavassori, Valentina; Nonis, Alessandro; Canarutto, Daniele; Naldini, Luigi.

Cell ; 185(13): 2248-2264.e21, 2022 06 23.

Artigo Inglês | MEDLINE | ID: mdl-35617958

10.

Of mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease.

Makani, Julie; Luzzatto, Lucio.

Cell ; 185(8): 1261-1265, 2022 04 14.

Artigo Inglês | MEDLINE | ID: mdl-35385685

11.

Adoptive immunotherapy for cancer or viruses.

Maus, Marcela V; Fraietta, Joseph A; Levine, Bruce L; Kalos, Michael; Zhao, Yangbing; June, Carl H.

Annu Rev Immunol ; 32: 189-225, 2014.

Artigo Inglês | MEDLINE | ID: mdl-24423116

12.

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors.

Wang, Dan; Zhang, Feng; Gao, Guangping.

Cell ; 181(1): 136-150, 2020 04 02.

Artigo Inglês | MEDLINE | ID: mdl-32243786

13.

Mitochondrial Diseases: Hope for the Future.

Russell, Oliver M; Gorman, Gráinne S; Lightowlers, Robert N; Turnbull, Doug M.

Cell ; 181(1): 168-188, 2020 04 02.

Artigo Inglês | MEDLINE | ID: mdl-32220313

14.

Epigenetic Therapy for Epithelioid Sarcoma.

Rothbart, Scott B; Baylin, Stephen B.

Cell ; 181(2): 211, 2020 04 16.

Artigo Inglês | MEDLINE | ID: mdl-32302562

15.

One Prime for All Editing.

Yang, Li; Yang, Bei; Chen, Jia.

Cell ; 179(7): 1448-1450, 2019 Dec 12.

Artigo Inglês | MEDLINE | ID: mdl-31835025

16.

The Future of Genome Editing.

Cell ; 173(6): 1311-1313, 2018 05 31.

Artigo Inglês | MEDLINE | ID: mdl-29856947

17.

Gene Therapy for Retinal Degeneration.

Apte, Rajendra S.

Cell ; 173(1): 5, 2018 03 22.

Artigo Inglês | MEDLINE | ID: mdl-29570997

18.

FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B.

Lillicrap, David.

Cell ; 171(7): 1478-1480, 2017 12 14.

Artigo Inglês | MEDLINE | ID: mdl-29245009

19.

CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes.

Komor, Alexis C; Badran, Ahmed H; Liu, David R.

Cell ; 168(1-2): 20-36, 2017 Jan 12.

Artigo Inglês | MEDLINE | ID: mdl-27866654

20.

Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9.

Batra, Ranjan; Nelles, David A; Pirie, Elaine; Blue, Steven M; Marina, Ryan J; Wang, Harrison; Chaim, Isaac A; Thomas, James D; Zhang, Nigel; Nguyen, Vu; Aigner, Stefan; Markmiller, Sebastian; Xia, Guangbin; Corbett, Kevin D; Swanson, Maurice S; Yeo, Gene W.

Cell ; 170(5): 899-912.e10, 2017 Aug 24.

Artigo Inglês | MEDLINE | ID: mdl-28803727

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