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Public Health Genomics and Precision Health Knowledge Base (v9.0)
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Precision Health Database|Search|Public Health Genomics and Precision Health Knowledge Base (PHGKB)
Rare Diseases
Last data update: Apr 25, 2024
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Beta-thalassemia[original query]>>Reviews/Commentaries[Product Type]
Gene therapy for Hemoglobinopathies.
Maria Rosa Lidonnici et al. Hum Gene Ther 2023
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Fetal Hemoglobin Regulation in Beta-Thalassemia.
Henry Y Lu et al. Hematology/oncology clinics of North America 2023 37(2) 301-312
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Non-Transfusion-Dependent Thalassemia: A Panoramic Review.
Shash Hwazen et al. Medicina (Kaunas, Lithuania) 2022 58(10)
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Gene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell Disease.
Leonard Alexis et al. Hematology/oncology clinics of North America 2022
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Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm.
Baronciani Donatella et al. HemaSphere 2021 5(5) e555
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A literature review on the parvovirus B19 infection in sickle cell anemia and β-thalassemia patients.
Soltani Saber et al. Tropical medicine and health 2020 Dec 48(1) 96
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CRISPR Approach To Fighting Cancer Called 'Promising' In 1st Safety Test
R Stein, NPR, November 6, 2019
Heritable Genome Editing: Who Speaks for "Future" Children?
Knoppers Bartha Maria et al. The CRISPR journal 2019 Oct 2(5) 285-292
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CRISPR enters its first human clinical trials- The gene editor targets cancer, blood disorders and blindness
T Hesman, Science News, August 14, 2019
Sickle Cell Patient Reveals Why She Is Volunteering For Landmark Gene-Editing Study
R Stein, NPR, July 29, 2019
Advances in the Gene Therapy of Monogenic Blood Cell Diseases.
Bueren Juan A et al. Clinical genetics 2019 Jun
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Validation of Extensive Next-Generation Sequencing Method for Monogenic Disorder Analysis on Cell Free Fetal DNA: Noninvasive Prenatal Diagnosis.
Dello Russo Claudio et al. The Journal of molecular diagnostics : JMD 2019 Apr
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Beta Thalassemia: Monitoring and New Treatment Approaches.
Khandros Eugene et al. Hematology/oncology clinics of North America 2019 Jun 33(3) 339-353
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Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges.
Ghiaccio Valentina et al. Molecular diagnosis & therapy 2019 Jan
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Innovation for rare diseases and bioethical concerns: A thin thread between medical progress and suffering.
Tommasini Alberto et al. World journal of clinical pediatrics 2018 Aug 7(3) 75-82
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Gene therapy reduces need for transfusions in severe blood disorder
NIH Research Matters, May 1, 2018
Gene therapy helps patients avoid blood transfusion, study says
CNN, Apr 19, 2018
A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.
Cai Liuhong et al. Stem cells translational medicine 2017 Nov
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Sickle Cell Trait: A Benign State?
Kotila Taiwo R et al. Acta haematologica 2016 136(3) 147-51
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Review: Beta-thalassemia and molecular chaperones.
Sumera Afshan et al. Blood Cells Mol. Dis. 2015 Apr (4) 348-352
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Page last reviewed:
Feb 1, 2024
Page last updated:
Apr 25, 2024
Content source:
Public Health Genomics Branch in the Division of Blood Disorders and Public Health Genomics
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National Center on Birth Defects and Developmental Disabilities
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