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Literature DB >> 24804755

CRISPR technology for gene therapy.

Katherine High, Philip D Gregory, Charles Gersbach.

Abstract

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Year: 2014 PMID： 24804755 DOI： 10.1038/nm.3566

Source DB: PubMed Journal: Nat Med ISSN： 1078-8956 Impact factor: 53.440

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12 in total

1. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors.

Authors: R O Snyder; C H Miao; G A Patijn; S K Spratt; O Danos; D Nagy; A M Gown; B Winther; L Meuse; L K Cohen; A R Thompson; M A Kay
Journal: Nat Genet Date: 1997-07 Impact factor: 38.330

2. An unbiased genome-wide analysis of zinc-finger nuclease specificity.

Authors: Richard Gabriel; Angelo Lombardo; Anne Arens; Jeffrey C Miller; Pietro Genovese; Christine Kaeppel; Ali Nowrouzi; Cynthia C Bartholomae; Jianbin Wang; Geoffrey Friedman; Michael C Holmes; Philip D Gregory; Hanno Glimm; Manfred Schmidt; Luigi Naldini; Christof von Kalle
Journal: Nat Biotechnol Date: 2011-08-07 Impact factor: 54.908

3. Correction of a genetic disease in mouse via use of CRISPR-Cas9.

Authors: Yuxuan Wu; Dan Liang; Yinghua Wang; Meizhu Bai; Wei Tang; Shiming Bao; Zhiqiang Yan; Dangsheng Li; Jinsong Li
Journal: Cell Stem Cell Date: 2013-12-05 Impact factor: 24.633

4. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients.

Authors: Gerald Schwank; Bon-Kyoung Koo; Valentina Sasselli; Johanna F Dekkers; Inha Heo; Turan Demircan; Nobuo Sasaki; Sander Boymans; Edwin Cuppen; Cornelis K van der Ent; Edward E S Nieuwenhuis; Jeffrey M Beekman; Hans Clevers
Journal: Cell Stem Cell Date: 2013-12-05 Impact factor: 24.633

Review 5. Cas9 as a versatile tool for engineering biology.

Authors: Prashant Mali; Kevin M Esvelt; George M Church
Journal: Nat Methods Date: 2013-10 Impact factor: 28.547

6. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.

Authors: Pablo Tebas; David Stein; Winson W Tang; Ian Frank; Shelley Q Wang; Gary Lee; S Kaye Spratt; Richard T Surosky; Martin A Giedlin; Geoff Nichol; Michael C Holmes; Philip D Gregory; Dale G Ando; Michael Kalos; Ronald G Collman; Gwendolyn Binder-Scholl; Gabriela Plesa; Wei-Ting Hwang; Bruce L Levine; Carl H June
Journal: N Engl J Med Date: 2014-03-06 Impact factor: 91.245

Review 7. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.

Authors: Thomas Gaj; Charles A Gersbach; Carlos F Barbas
Journal: Trends Biotechnol Date: 2013-05-09 Impact factor: 19.536

8. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

Authors: Hao Yin; Wen Xue; Sidi Chen; Roman L Bogorad; Eric Benedetti; Markus Grompe; Victor Koteliansky; Phillip A Sharp; Tyler Jacks; Daniel G Anderson
Journal: Nat Biotechnol Date: 2014-03-30 Impact factor: 54.908

9. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Authors: Amit C Nathwani; Edward G D Tuddenham; Savita Rangarajan; Cecilia Rosales; Jenny McIntosh; David C Linch; Pratima Chowdary; Anne Riddell; Arnulfo Jaquilmac Pie; Chris Harrington; James O'Beirne; Keith Smith; John Pasi; Bertil Glader; Pradip Rustagi; Catherine Y C Ng; Mark A Kay; Junfang Zhou; Yunyu Spence; Christopher L Morton; James Allay; John Coleman; Susan Sleep; John M Cunningham; Deokumar Srivastava; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; John T Gray; Ulrike M Reiss; Arthur W Nienhuis; Andrew M Davidoff
Journal: N Engl J Med Date: 2011-12-10 Impact factor: 176.079

10. In vivo genome editing restores haemostasis in a mouse model of haemophilia.

Authors: Hojun Li; Virginia Haurigot; Yannick Doyon; Tianjian Li; Sunnie Y Wong; Anand S Bhagwat; Nirav Malani; Xavier M Anguela; Rajiv Sharma; Lacramiora Ivanciu; Samuel L Murphy; Jonathan D Finn; Fayaz R Khazi; Shangzhen Zhou; David E Paschon; Edward J Rebar; Frederic D Bushman; Philip D Gregory; Michael C Holmes; Katherine A High
Journal: Nature Date: 2011-06-26 Impact factor: 49.962

12 in total

1. In vivo gene therapy potentials of CRISPR-Cas9.

Authors: H-Y Xue; X Zhang; Y Wang; L Xiaojie; W-J Dai; Y Xu
Journal: Gene Ther Date: 2016-03-31 Impact factor: 5.250

2. Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells.

Authors: Yuxuan Wu; Hai Zhou; Xiaoying Fan; Ying Zhang; Man Zhang; Yinghua Wang; Zhenfei Xie; Meizhu Bai; Qi Yin; Dan Liang; Wei Tang; Jiaoyang Liao; Chikai Zhou; Wujuan Liu; Ping Zhu; Hongshan Guo; Hong Pan; Chunlian Wu; Huijuan Shi; Ligang Wu; Fuchou Tang; Jinsong Li
Journal: Cell Res Date: 2014-12-05 Impact factor: 25.617

CRISPR technology for gene therapy.

1. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors.

2. An unbiased genome-wide analysis of zinc-finger nuclease specificity.

3. Correction of a genetic disease in mouse via use of CRISPR-Cas9.

4. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients.

Review 5. Cas9 as a versatile tool for engineering biology.

6. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.

Review 7. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.

8. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

9. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

10. In vivo genome editing restores haemostasis in a mouse model of haemophilia.

1. In vivo gene therapy potentials of CRISPR-Cas9.

2. Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells.

Review 3. Epigenome engineering in cancer: fairytale or a realistic path to the clinic?

Review 4. Cellular reprogramming for understanding and treating human disease.

5. Design of a colicin E7 based chimeric zinc-finger nuclease.

Review 6. Phosphorylation of the regulatory light chain of myosin in striated muscle: methodological perspectives.

7. Expression of PTEN-long mediated by CRISPR/Cas9 can repress U87 cell proliferation.

8. Bioinformatic prediction and functional characterization of human KIAA0100 gene.

9. Enhanced non-viral gene delivery by coordinated endosomal release and inhibition of β-tubulin deactylase.

10. CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation.