Literature DB >> 28190778

Genome Editing for Sickle Cell Disease: A Little BCL11A Goes a Long Way.

Mir A Hossain1, Jörg Bungert2.   

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Year:  2017        PMID： 28190778      PMCID： PMC5363208          DOI： 10.1016/j.ymthe.2017.02.003

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  9 in total

1.  Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.

Authors:  Mark A DeWitt; Wendy Magis; Nicolas L Bray; Tianjiao Wang; Jennifer R Berman; Fabrizia Urbinati; Seok-Jin Heo; Therese Mitros; Denise P Muñoz; Dario Boffelli; Donald B Kohn; Mark C Walters; Dana Carroll; David I K Martin; Jacob E Corn
Journal:  Sci Transl Med       Date:  2016-10-12       Impact factor: 17.956

2.  Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia.

Authors:  Lin Ye; Jiaming Wang; Yuting Tan; Ashley I Beyer; Fei Xie; Marcus O Muench; Yuet Wai Kan
Journal:  Proc Natl Acad Sci U S A       Date:  2016-09-06       Impact factor: 11.205

3.  Strict in vivo specificity of the Bcl11a erythroid enhancer.

Authors:  Elenoe C Smith; Sidinh Luc; Donyell M Croney; Mollie B Woodworth; Luciano C Greig; Yuko Fujiwara; Minh Nguyen; Falak Sher; Jeffrey D Macklis; Daniel E Bauer; Stuart H Orkin
Journal:  Blood       Date:  2016-10-05       Impact factor: 22.113

4.  Rationally engineered Cas9 nucleases with improved specificity.

Authors:  Ian M Slaymaker; Linyi Gao; Bernd Zetsche; David A Scott; Winston X Yan; Feng Zhang
Journal:  Science       Date:  2015-12-01       Impact factor: 47.728

5.  CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

Authors:  Daniel P Dever; Rasmus O Bak; Andreas Reinisch; Joab Camarena; Gabriel Washington; Carmencita E Nicolas; Mara Pavel-Dinu; Nivi Saxena; Alec B Wilkens; Sruthi Mantri; Nobuko Uchida; Ayal Hendel; Anupama Narla; Ravindra Majeti; Kenneth I Weinberg; Matthew H Porteus
Journal:  Nature       Date:  2016-11-07       Impact factor: 49.962

6.  BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis.

Authors:  Matthew C Canver; Elenoe C Smith; Falak Sher; Luca Pinello; Neville E Sanjana; Ophir Shalem; Diane D Chen; Patrick G Schupp; Divya S Vinjamur; Sara P Garcia; Sidinh Luc; Ryo Kurita; Yukio Nakamura; Yuko Fujiwara; Takahiro Maeda; Guo-Cheng Yuan; Feng Zhang; Stuart H Orkin; Daniel E Bauer
Journal:  Nature       Date:  2015-09-16       Impact factor: 49.962

7.  Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.

Authors:  Kai-Hsin Chang; Sarah E Smith; Timothy Sullivan; Kai Chen; Qianhe Zhou; Jason A West; Mei Liu; Yingchun Liu; Benjamin F Vieira; Chao Sun; Vu P Hong; Mingxuan Zhang; Xiao Yang; Andreas Reik; Fyodor D Urnov; Edward J Rebar; Michael C Holmes; Olivier Danos; Haiyan Jiang; Siyuan Tan
Journal:  Mol Ther Methods Clin Dev       Date:  2017-01-11       Impact factor: 6.698

8.  A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.

Authors:  Elizabeth A Traxler; Yu Yao; Yong-Dong Wang; Kaitly J Woodard; Ryo Kurita; Yukio Nakamura; Jim R Hughes; Ross C Hardison; Gerd A Blobel; Chunliang Li; Mitchell J Weiss
Journal:  Nat Med       Date:  2016-08-15       Impact factor: 53.440

9.  High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.

Authors:  Benjamin P Kleinstiver; Vikram Pattanayak; Michelle S Prew; Shengdar Q Tsai; Nhu T Nguyen; Zongli Zheng; J Keith Joung
Journal:  Nature       Date:  2016-01-06       Impact factor: 49.962
  9 in total
  2 in total

1.  Fetal hemoglobin induction in sickle erythroid progenitors using a synthetic zinc finger DNA-binding domain.

Authors:  Biaoru Li; Xingguo Zhu; Mir A Hossain; Cameron R Guy; Hongyan Xu; Jörg Bungert; Betty S Pace
Journal:  Haematologica       Date:  2018-04-05       Impact factor: 9.941

Review 2.  The state of gene therapy research in Africa, its significance and implications for the future.

Authors:  P Arbuthnot; M B Maepa; A Ely; M S Pepper
Journal:  Gene Ther       Date:  2017-07-10       Impact factor: 5.250
  2 in total

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