The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation (Q45870741)

17 December 2017

title

The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation (English)

1 reference

17 December 2017

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Daniel P Dever

1

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17 December 2017

Matthew H Porteus

series ordinal

2

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17 December 2017

publication date

12 August 2017

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17 December 2017

Current Opinion in Hematology

published in

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17 December 2017

volume

24

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17 December 2017

issue

6

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17 December 2017

page(s)

481-488

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17 December 2017

Marker-free coselection for CRISPR-driven genome editing in human cells.

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In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery

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

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

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CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

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Genome Editing: A New Approach to Human Therapeutics

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Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

26 August 2018

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Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.

26 August 2018

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Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

26 August 2018

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BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

26 August 2018

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Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

26 August 2018

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

26 August 2018

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Common severe infections in chronic granulomatous disease

26 August 2018

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Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9

26 August 2018

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Targeted genome editing in human repopulating haematopoietic stem cells

26 August 2018

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An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level.

26 August 2018

1 reference

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A functional element necessary for fetal hemoglobin silencing

26 August 2018

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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

26 August 2018

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

26 August 2018

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

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Gene therapy: therapeutic gene causing lymphoma

26 August 2018

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Chimeric nucleases stimulate gene targeting in human cells

26 August 2018

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Bcl11a is essential for normal lymphoid development

26 August 2018

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

26 August 2018

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Management of sickle cell disease.

26 August 2018

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Molecular basis of hereditary persistence of fetal hemoglobin

26 August 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5766279

Increased fetal hemoglobin in patients receiving sodium 4-phenylbutyrate

26 August 2018

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12 December 2020

Allogeneic marrow transplantation for thalassemia

1 reference

12 December 2020

Bone-marrow transplantation in a patient with sickle-cell anemia

1 reference

12 December 2020

Immunological reconstitution of sex-linked lymphopenic immunological deficiency

1 reference

12 December 2020

10.1097/MOH.0000000000000385

Identifiers

DOI

1 reference

17 December 2017

0 references

1 reference

17 December 2017