(Q57171107)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30070404%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

title

Treatment of β654 -thalassaemia by TALENs in a mouse model (English)

1 reference

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15 April 2020

11

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15 April 2020

author name string

Yudan Fang

1

1 reference

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15 April 2020

Yan Cheng

2

1 reference

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15 April 2020

Dan Lu

3

1 reference

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15 April 2020

Xiuli Gong

4

1 reference

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15 April 2020

Guanheng Yang

5

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15 April 2020

Zhijuan Gong

6

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15 April 2020

Yiwen Zhu

7

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15 April 2020

Xiao Sang

8

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15 April 2020

Shuyue Fan

9

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15 April 2020

Jingzhi Zhang

10

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15 April 2020

language of work or name

English

0 references

publication date

2 August 2018

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15 April 2020

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15 April 2020

volume

51

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15 April 2020

issue

6

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15 April 2020

page(s)

e12491

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Detection of beta-thalassemia mutations in the Chinese using amplified DNA from dried blood specimens

15 April 2020

cites work

1 reference

12 December 2020

beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects

1 reference

12 December 2020

RNA transcripts of the beta-thalassaemia allele IVS-2-654 C-->T: a small amount of normally processed beta-globin mRNA is still produced from the mutant gene

1 reference

12 December 2020

Survival in medically treated patients with homozygous beta-thalassemia

1 reference

12 December 2020

A common human beta globin splicing mutation modeled in mice.

1 reference

12 December 2020

Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing.

1 reference

12 December 2020

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia

1 reference

12 December 2020

Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach

1 reference

12 December 2020

High-efficiency thermal asymmetric interlaced PCR for amplification of unknown flanking sequences

1 reference

12 December 2020

A novel transgenic mouse model produced from lentiviral germline integration for the study of beta-thalassemia gene therapy

1 reference

12 December 2020

Global epidemiology of haemoglobin disorders and derived service indicators

1 reference

12 December 2020

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

1 reference

12 December 2020

Amelioration of beta654-thalassemia in mouse model with the knockdown of aberrantly spliced beta-globin mRNA.

1 reference

12 December 2020

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

1 reference

12 December 2020

Transgenic human alpha-hemoglobin stabilizing protein could partially relieve betaIVS-2-654-thalassemia syndrome in model mice

1 reference

12 December 2020

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

1 reference

12 December 2020

Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors

1 reference

12 December 2020

Genetic engineering of human pluripotent cells using TALE nucleases

1 reference

12 December 2020

TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: tools for TAL effector design and target prediction

1 reference

12 December 2020

Multiplex genome engineering using CRISPR/Cas systems

1 reference

12 December 2020

RNA-guided human genome engineering via Cas9

1 reference

12 December 2020

Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs

1 reference

12 December 2020

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

1 reference

12 December 2020

Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells

1 reference

12 December 2020

Nuclease-mediated gene editing by homologous recombination of the human globin locus

1 reference

12 December 2020

A guide to genome engineering with programmable nucleases

1 reference

12 December 2020

Therapeutic effects of induced pluripotent stem cells in chimeric mice with β-thalassemia

1 reference

12 December 2020

Targeted genome editing in human repopulating haematopoietic stem cells

1 reference

12 December 2020

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac

1 reference

12 December 2020

Efficient and allele-specific genome editing of disease loci in human iPSCs

1 reference

12 December 2020

Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system

1 reference

12 December 2020

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

1 reference

12 December 2020

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs

1 reference

12 December 2020

Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells

1 reference

12 December 2020

A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

1 reference

12 December 2020

Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World.

1 reference

12 December 2020

Identifiers

DOI

10.1111/CPR.12491

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30070404%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

1 reference

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15 April 2020

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