(Q40412263)

20 September 2017

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells (English)

1 reference

20 September 2017

0 references

Mark A DeWitt

1

1 reference

20 September 2017

Wendy Magis

2

1 reference

20 September 2017

Nicolas L Bray

3

1 reference

20 September 2017

Tianjiao Wang

4

1 reference

20 September 2017

Jennifer R Berman

5

1 reference

20 September 2017

Fabrizia Urbinati

6

1 reference

20 September 2017

Seok-Jin Heo

7

1 reference

20 September 2017

Therese Mitros

8

1 reference

20 September 2017

Denise P Muñoz

9

1 reference

20 September 2017

Dario Boffelli

10

1 reference

20 September 2017

Donald B Kohn

11

1 reference

20 September 2017

Mark C Walters

12

1 reference

20 September 2017

Dana Carroll

13

1 reference

20 September 2017

David I K Martin

14

1 reference

20 September 2017

Jacob E Corn

15

1 reference

20 September 2017

language of work or name

English

0 references

publication date

1 October 2016

1 reference

Science Translational Medicine

20 September 2017

1 reference

20 September 2017

8

1 reference

20 September 2017

360

1 reference

20 September 2017

360ra134

1 reference

Non-homologous DNA increases gene disruption efficiency by altering DNA repair outcomes.

20 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Near-optimal probabilistic RNA-seq quantification

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Rationally engineered Cas9 nucleases with improved specificity

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Management of sickle cell disease: a review for physician education in Nigeria (sub-saharan Africa).

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Rational design of highly active sgRNAs for CRISPR-Cas9-mediated gene inactivation.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

A guide to genome engineering with programmable nucleases

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

DNA targeting specificity of RNA-guided Cas9 nucleases

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

RNA-programmed genome editing in human cells

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Engineering humanized mice for improved hematopoietic reconstitution

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

In vivo genome editing restores haemostasis in a mouse model of haemophilia

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Mendelian proportions in a mixed population

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Evidence for ineffective erythropoiesis in severe sickle cell disease

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Ex vivo generation of fully mature human red blood cells from hematopoietic stem cells

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Sickle cell anemia, a molecular disease

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

The human genome browser at UCSC

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Genome editing technologies: defining a path to clinic

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Targeted genome editing in human repopulating haematopoietic stem cells

15 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

The effect of fetal hemoglobin on the survival characteristics of sickle cells

15 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection.

15 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

TALEN-mediated generation and genetic correction of disease-specific human induced pluripotent stem cells

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Isolation of single human hematopoietic stem cells capable of long-term multilineage engraftment.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

The Inheritance of Sickle Cell Anemia.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Mortality in sickle cell disease. Life expectancy and risk factors for early death.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500303

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA.

20 August 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF9336

RSEM: accurate transcript quantification from RNA-Seq data with or without a reference genome

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF9336

Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF9336

Effects of mixed hematopoietic chimerism in a mouse model of bone marrow transplantation for sickle cell anemia

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27733558

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCITRANSLMED.AAF9336

1 reference

20 September 2017

1 reference

20 September 2017

1 reference