Literature DB >> 29699995

A chance to cut (the genome) is a chance to cure.

Kara E Montbleau1, Vijay G Sankaran1.   

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Year:  2018        PMID： 29699995      PMCID： PMC5921966          DOI： 10.1182/blood-2018-03-839787

Source DB:  PubMed          Journal:  Blood        ISSN： 0006-4971            Impact factor:   22.113


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  10 in total

1.  Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A.

Authors:  Vijay G Sankaran; Tobias F Menne; Jian Xu; Thomas E Akie; Guillaume Lettre; Ben Van Handel; Hanna K A Mikkola; Joel N Hirschhorn; Alan B Cantor; Stuart H Orkin
Journal:  Science       Date:  2008-12-04       Impact factor: 47.728

2.  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

3.  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

4.  Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

Authors:  Chiara Antoniani; Vasco Meneghini; Annalisa Lattanzi; Tristan Felix; Oriana Romano; Elisa Magrin; Leslie Weber; Giulia Pavani; Sara El Hoss; Ryo Kurita; Yukio Nakamura; Thomas J Cradick; Ante S Lundberg; Matthew Porteus; Mario Amendola; Wassim El Nemer; Marina Cavazzana; Fulvio Mavilio; Annarita Miccio
Journal:  Blood       Date:  2018-03-08       Impact factor: 22.113

Review 5.  Anemia: progress in molecular mechanisms and therapies.

Authors:  Vijay G Sankaran; Mitchell J Weiss
Journal:  Nat Med       Date:  2015-03       Impact factor: 53.440

6.  A functional element necessary for fetal hemoglobin silencing.

Authors:  Vijay G Sankaran; Jian Xu; Rachel Byron; Harvey A Greisman; Chris Fisher; David J Weatherall; Daniel E Sabath; Mark Groudine; Stuart H Orkin; Anuja Premawardhena; M A Bender
Journal:  N Engl J Med       Date:  2011-09-01       Impact factor: 91.245

7.  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

8.  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

9.  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

10.  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
  10 in total

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