CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease. (Q40375767)

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9 April 2020

title

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

1 reference

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X-linked chronic granulomatous disease

9 April 2020

main subject

CRISPR

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18

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9 April 2020

Harry Malech

20

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9 April 2020

1

Suk See De Ravin

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9 April 2020

17

Douglas Kuhns

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9 April 2020

4

Uimook Choi

1 reference

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9 April 2020

11

Colin Sweeney

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9 April 2020

6

Sherry Koontz

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9 April 2020

19

Madhusudan V Peshwa

1 reference

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9 April 2020

12

Lela Kardava

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9 April 2020

author name string

Linhong Li

2

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9 April 2020

Xiaolin Wu

3

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9 April 2020

Cornell Allen

5

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9 April 2020

Janet Lee

7

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9 April 2020

Narda Theobald-Whiting

8

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9 April 2020

Jessica Chu

9

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9 April 2020

Mary Garofalo

10

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9 April 2020

Angelia Viley

14

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9 April 2020

Pachai Natarajan

15

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9 April 2020

Ling Su

16

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9 April 2020

Susan Moir

13

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9 April 2020

language of work or name

English

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publication date

1 January 2017

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Science Translational Medicine

9 April 2020

published in

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9 April 2020

volume

9

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9 April 2020

issue

372

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

9 April 2020

cites work

1 reference

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

21 January 2018

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

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

21 January 2018

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

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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

21 January 2018

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

21 January 2018

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A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

21 January 2018

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Cas9 as a versatile tool for engineering biology

21 January 2018

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Multiplex genome engineering using CRISPR/Cas systems

21 January 2018

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Genome editing in rice and wheat using the CRISPR/Cas system.

21 January 2018

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

21 January 2018

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

21 January 2018

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Targeted genome editing in human repopulating haematopoietic stem cells

21 January 2018

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Residual NADPH oxidase and survival in chronic granulomatous disease

21 January 2018

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

21 January 2018

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

21 January 2018

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Gene editing using ssODNs with engineered endonucleases.

21 January 2018

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Stable gene targeting in human cells using single-strand oligonucleotides with modified bases

21 January 2018

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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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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21 January 2018

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21 January 2018

Identifiers

DOI

1 reference

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9 April 2020

1 reference