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CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.

author

Kol A Zarember

Harry Malech

Suk See De Ravin

Douglas B Kuhns

Uimook Choi

Colin L Sweeney

Sherry Koontz

Madhusudan V Peshwa

Lela Kardava

published in: Science Translational Medicine

date of publication: 2017-01-01

language: English

main subject: CRISPR, X-linked chronic granulomatous disease, Cas9

Cites articles 29

Date

Title

Authors

2016-04-01

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Kol A Zarember, Geraldine M O'Connor, Harry Malech, Luigi D Notarangelo, Suk See De Ravin, Douglas B Kuhns, Uimook Choi, Elizabeth Kang, Narda Theobald, Lela Kardava
2016-06-02

AQP8 transports NOX2-generated H2O2 across the plasma membrane to promote signaling in B cells

Roberto Sitia, Mauro Galli, Alessandro Aiuti
2016-01-06

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

Benjamin P Kleinstiver, Zongli Zheng, J. Keith Joung
2015-12-09

An assessment of the effects of ectopic gp91phox expression in XCGD iPSC-derived neutrophils

Mahito Nakanishi
2015-03-02

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Gregory J Cost, Aaron Cooper, Andrew Wilber, Donald B. Kohn, Megan D Hoban, Zulema Romero, Michael L Kaufman, Michelle Ho, David Gray, Georgia R Lill, Philip D Gregory, Edward Rebar, David E Paschon, Andreas Reik, Pei-Qi Liu
2015-06-19

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

Walther Hänseler, William James, Sally A. Cowley
2015-01-01

Gene editing using ssODNs with engineered endonucleases.
2015-11-02

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

Benjamin P Kleinstiver, Zongli Zheng, J. Keith Joung
2015-06-22

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

Benjamin P Kleinstiver, Zongli Zheng, Martin J. Aryee, J. Keith Joung
2014-08-07

Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency

Tayfun Güngör, Morton J Cowan, Luigi D Notarangelo, Petr Sedláček, Andrew R Gennery, Arjan Lankester, Christopher C Dvorak, Rebecca H. Buckley, Michael A Pulsipher, Nancy J Bunin, Paul Vey-Hong Lim, Jacob Bleesing
2014-09-18

Genome editing in rice and wheat using the CRISPR/Cas system.
2014-11-28

Genome editing. The new frontier of genome engineering with CRISPR-Cas9

Emmanuelle Charpentier, Jennifer Doudna
2014-05-28

Targeted genome editing in human repopulating haematopoietic stem cells

Pietro Genovese, Giulia Schiroli, Giulia Escobar, Andrea Calabria, Roberta Mazzieri, Maria Chiara Bonini, Philip D Gregory, Bernhard Gentner, Eugenio Montini, Angelo Leone Lombardo, Luigi Naldini
2014-08-21

Mobilization characteristics and strategies to improve hematopoietic progenitor cell mobilization and collection in patients with chronic granulomatous disease and severe combined immunodeficiency

Harry Malech, Suk See De Ravin
2014-05-29

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis

Erika Zonari, Ezio Giorda, Anna Kajaste-Rudnitski, Paolo Rossi, Andrea Finocchi, Luigi Naldini, Bernhard Gentner, Alessandro Aiuti
2013-07-11

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

Elia Stupka, Christof von Kalle, Luigi Naldini, Francesca Fumagalli, Andrea Calabria, Eugenio Montini, Sabata Martino, Jaap J. Boelens, Alessandro Aiuti, Maria Grazia Roncarolo, Claudio Bordignon, Gianluigi Zanetti, Manfred Schmidt, Fabio Ciceri, Tiziana Plati, Maria Sessa, Clelia Di Serio
2013-07-11

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

Luigi Naldini, Francesca Ferrua, Andrea Calabria, Maria Carmina Castiello, Eugenio Montini, Andrea Finocchi, Alessandro Aiuti, Jordan Orange, Anna Villa, Maria Grazia Roncarolo, Christof Von Kalle, Manfred Schmidt, Fabio Ciceri, Ayşe Metin, Anne Galy, Clelia Di Serio
2013-10

Cas9 as a versatile tool for engineering biology

Kevin M. Esvelt, George M. Church
2013-02-15

Multiplex genome engineering using CRISPR/Cas systems

Xuebing Wu, Patrick D Hsu
2013-11-01

Clinical manifestations of disease in X-linked carriers of chronic granulomatous disease

David Goldblatt
2012-06-28

A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity

Martin Jínek, Krzysztof Chylinski, Ines Fonfara, Michael Hauer, Jennifer Doudna, Emmanuelle Charpentier
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Stable gene targeting in human cells using single-strand oligonucleotides with modified bases

George M. Church, Adrian W. Briggs
2012-10-16

Humans with chronic granulomatous disease maintain humoral immunologic memory despite low frequencies of circulating memory B cells

Anthony Fauci, Brian H Santich, Harry Malech, Tae-Wook Chun, Susan Moir, Suk See De Ravin
2011-12-19

Innate immunity against Granulibacter bethesdensis, an emerging gram-negative bacterial pathogen

Michael E Fenster, Jessica Chu, Frank R. DeLeo, Harry Malech, Kol A Zarember, John I. Gallin, Adam R Shoffner, Kimberly R Marshall-Batty
2010-11-01

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

Christof von Kalle, Jordan Orange, Ricardo A Dewey, Claudia R Ball, Manfred Schmidt
2010-12-30

Residual NADPH oxidase and survival in chronic granulomatous disease

Harry Malech, Kol A Zarember, Gulbu Uzel, Douglas B Kuhns, John I. Gallin, Debra A Long Priel, Theo Heller, Beatriz E Marciano
2010-10-26

Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells

Elena Almarza, Juan Bueren, Harry Malech, Adrian Thrasher
2009-03-01

Use of internally nuclease-protected single-strand DNA oligonucleotides and silencing of the mismatch repair protein, MSH2, enhances the replication of corrected cells following gene editing
2006-04-02

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

Christof von Kalle, Stefan Stein, Claudia R Ball, Ulrike Koehl, Moustapha Hassan, Cynthia E. Dunbar, Adrian Thrasher, Neal G. Copeland, Nancy A. Jenkins, Manfred Schmidt, Manuel Grez, Kerstin Schwarzwaelder, Hanno Glimm

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