Literature DB >> 28109956

A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).

Giorgia Santilli1, Adrian J Thrasher2.   

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Year:  2017        PMID: 28109956      PMCID: PMC5368843          DOI: 10.1016/j.ymthe.2017.01.002

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


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  14 in total

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

Authors:  Colin L Sweeney; Jizhong Zou; Uimook Choi; Randall K Merling; Alexander Liu; Aaron Bodansky; Sandra Burkett; Jung-Woong Kim; Suk See De Ravin; Harry L Malech
Journal:  Mol Ther       Date:  2017-02-01       Impact factor: 11.454

2.  Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease.

Authors:  I Barde; E Laurenti; S Verp; M Wiznerowicz; S Offner; A Viornery; A Galy; A Trumpp; D Trono
Journal:  Gene Ther       Date:  2011-05-05       Impact factor: 5.250

Review 3.  Molecular diagnosis of chronic granulomatous disease.

Authors:  D Roos; M de Boer
Journal:  Clin Exp Immunol       Date:  2014-02       Impact factor: 4.330

4.  Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting.

Authors:  Jizhong Zou; Colin L Sweeney; Bin-Kuan Chou; Uimook Choi; Jason Pan; Hongmei Wang; Sarah N Dowey; Linzhao Cheng; Harry L Malech
Journal:  Blood       Date:  2011-03-16       Impact factor: 22.113

5.  CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells.

Authors:  Rowan Flynn; Alexander Grundmann; Peter Renz; Walther Hänseler; William S James; Sally A Cowley; Michael D Moore
Journal:  Exp Hematol       Date:  2015-06-19       Impact factor: 3.084

6.  Nucleosomes impede Cas9 access to DNA in vivo and in vitro.

Authors:  Max A Horlbeck; Lea B Witkowsky; Benjamin Guglielmi; Joseph M Replogle; Luke A Gilbert; Jacqueline E Villalta; Sharon E Torigoe; Robert Tjian; Jonathan S Weissman
Journal:  Elife       Date:  2016-03-17       Impact factor: 8.140

7.  Functional Restoration of gp91phox-Oxidase Activity by BAC Transgenesis and Gene Targeting in X-linked Chronic Granulomatous Disease iPSCs.

Authors:  Magdalena Laugsch; Maria Rostovskaya; Sergiy Velychko; Cornelia Richter; Ariane Zimmer; Barbara Klink; Evelin Schröck; Michael Haase; Katrin Neumann; Sebastian Thieme; Joachim Roesler; Sebastian Brenner; Konstantinos Anastassiadis
Journal:  Mol Ther       Date:  2015-08-28       Impact factor: 11.454

8.  Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects.

Authors:  Tamara J Laskowski; Yasmine Van Caeneghem; Rasoul Pourebrahim; Chao Ma; Zhenya Ni; Zita Garate; Ana M Crane; Xuan Shirley Li; Wei Liao; Manuel Gonzalez-Garay; Jose Carlos Segovia; David E Paschon; Edward J Rebar; Michael C Holmes; Dan Kaufman; Bart Vandekerckhove; Brian R Davis
Journal:  Stem Cell Reports       Date:  2016-07-07       Impact factor: 7.765

Review 9.  Genome-editing Technologies for Gene and Cell Therapy.

Authors:  Morgan L Maeder; Charles A Gersbach
Journal:  Mol Ther       Date:  2016-01-12       Impact factor: 11.454

10.  Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.

Authors:  Suk See De Ravin; Andreas Reik; Pei-Qi Liu; Linhong Li; Xiaolin Wu; Ling Su; Castle Raley; Narda Theobald; Uimook Choi; Alexander H Song; Andy Chan; Jocelynn R Pearl; David E Paschon; Janet Lee; Hannah Newcombe; Sherry Koontz; Colin Sweeney; David A Shivak; Kol A Zarember; Madhusudan V Peshwa; Philip D Gregory; Fyodor D Urnov; Harry L Malech
Journal:  Nat Biotechnol       Date:  2016-03-07       Impact factor: 54.908

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  1 in total

1.  A case-control study on correlation between the single nucleotide polymorphism of CLEC4E and the susceptibility to tuberculosis among Han people in Western China.

Authors:  Wenjing Zhou; Lijuan Wu; Jiajia Song; Lin Jiao; Yi Zhou; Juan Zhou; Nian Wang; Tangyuheng Liu; Zhenzhen Zhao; Hao Bai; Tao Wu; Binwu Ying
Journal:  BMC Infect Dis       Date:  2021-08-10       Impact factor: 3.090

  1 in total

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