Gene Therapy for Hemoglobinopathies

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Gene Therapy for Hemoglobinopathies

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description

article

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wetenschappelijk artikel

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наукова стаття, опублікована в жовтні 2018

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Gene Therapy for Hemoglobinopathies

@en

Gene Therapy for Hemoglobinopathies

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Gene Therapy for Hemoglobinopathies

@en

Gene Therapy for Hemoglobinopathies

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Gene Therapy for Hemoglobinopathies

@en

Gene Therapy for Hemoglobinopathies

@nl

P1476

Gene Therapy for Hemoglobinopathies

@en

P2093

Fulvio Mavilio

http://www.w3.org/2001/XMLSchema#string

Marina Cavazzana

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P275

Creative Commons Attribution

P2860

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

Retroviral integrations in gene therapy trials

The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene

Beta-thalassemia

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients

Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin.

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.

β-globin gene transfer to human bone marrow for sickle cell disease.

Correction of sickle cell disease in transgenic mouse models by gene therapy.

P304

1106-1113

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P31

scholarly article

P356

10.1089/HUM.2018.122

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P407

English

P433

http://www.w3.org/2001/XMLSchema#string

P478

http://www.w3.org/2001/XMLSchema#string

P50

Marina Cavazzana-Calvo

Fulvio Mavilio

P577

2018-10-01T00:00:00Z

http://www.w3.org/2001/XMLSchema#dateTime

P698

30200783

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P921

hemoglobinopathy

molecular medicine

blood proteins

biomedical investigative technique

molecular biology

hemeprotein

P932

6196755

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Gene Therapy for Hemoglobinopathies

about

Gene Therapy for Hemoglobinopathies

description

name

type

label

prefLabel

P1433

P1476

P2093

P275

P2860

P304

P31

P356

P407

P433

P478

P50

P577

P698

P921

P932

P356

P1433

P1476

P2093

P275

P2860

P304

P31

P356

P407

P433

P478

P50

P577

P698

P921

P932