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Public Health Genomics and Precision Health Knowledge Base (v9.0)
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Last data update: May 03, 2024
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Outcomes and long-term effects of hematopoietic stem cell transplant in sickle cell disease.
Zaina Inam et al. Expert Rev Hematol 2023
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Retrospective analysis of patients with severe combined immunodeficiency and alternative diagnostic criteria: A 20-year single centre experience.
Sevim Busra Korkmaz et al. Int J Immunogenet 2023
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Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials.
Eva Eugenie Rose Segura et al. Viruses 2023 15(3)
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A systematic review comparing allogeneic hematopoietic stem cell transplant to gene therapy in sickle cell disease.
Lianne E Rotin et al. Hematology (Amsterdam, Netherlands) 2023 28(1) 2163357
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Long-term health outcomes following curative therapies for sickle cell disease.
Chakravarthy Rohini et al. Hematology. American Society of Hematology. Education Program 2022 2022(1) 272-276
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Impact of Genetic Diagnosis on the Outcome of Hematopoietic Stem Cell Transplant in Primary Immunodeficiency Disorders.
Forlanini Federica et al. Journal of clinical immunology 2022
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Creating an Automated Contemporaneous Cohort in Sickle Cell Anemia to Predict Survival After Disease-Modifying Therapy.
Cronin Robert M et al. Blood advances 2022
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Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyond.
Egesa Walufu Ivan et al. International journal of pediatrics 2022 20223885979
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Umbilical cord blood as a hematopoietic stem cell source in transplantation for pediatric sickle cell disease: Current challenges and strategies.
Malhotra Megha et al. Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis 2022 103554
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Sickle Cell Disease: A Review.
Kavanagh Patricia L et al. JAMA 2022 328(1) 57-68
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Advances in the Management of Sickle Cell Disease: New Concepts and Future Horizons.
Higgins Tara et al. The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG 2022 27(3) 206-213
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Advances in the diagnosis and treatment of sickle cell disease.
Brandow A M et al. Journal of hematology & oncology 2022 15(1) 20
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Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Immunity.
Gonvindaraj Geeta M et al. Indian pediatrics 2021 58(2) 179-180
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A Decade Experience on Severe Combined Immunodeficiency Phenotype in Oman, Bridging to Newborn Screening.
Al Sukaiti Nashat et al. Frontiers in immunology 2020 11623199
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A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy.
Badawy Sherif M et al. Blood advances 2021 Jan 5(2) 570-583
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Clinical utility of 24-h rapid trio-exome sequencing for critically ill infants.
Wang Huijun et al. NPJ genomic medicine 2020 520
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Neonatal Mass Urine Screening Approach for Early Detection of Mucopolysaccharidoses by UPLC-MS/MS.
Menkovic Iskren et al. Diagnostics (Basel, Switzerland) 2019 Nov 9(4)
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Prepregnancy Genetic Carrier Screening in Recipients of Hematopoietic Stem Cell Transplants.
Aluko Ashley et al. Obstetrics and gynecology 2019 Sep
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Emerging non-genetic therapies for sickle cell disease.
Carden Marcus A et al. Haematologica 2019 Aug
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The North Carolina Experience with Mucopolysaccharidosis Type I Newborn Screening.
Taylor Jennifer L et al. The Journal of pediatrics 2019 May
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Pediatric Outcomes in Transplant: PersOnaliSing Immunosuppression To ImproVe Efficacy (POSITIVE Study): The Collaboration and Design of a National Transplant Precision Medicine Program.
Papaz Tanya et al. Transplantation direct 2018 Dec 4(12) e410
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Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries.
Fernandes Juliana Folloni et al. Journal of clinical immunology 2018 Nov
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Genetic Therapies for Sickle Cell Disease.
Jayavaradhan Rajeswari et al. Pediatric clinics of North America 2018 Jun 65(3) 465-480
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Family History of Early Infant Death Correlates with Earlier Age at Diagnosis But Not Shorter Time to Diagnosis for Severe Combined Immunodeficiency.
Luk Anderson Dik Wai et al. Frontiers in immunology 2017 8808
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Pediatric Sickle Cell Disease - Past Successes and Future Challenges.
Meier Emily Riehm et al. Pediatric research 2016 Oct
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Guidelines for Screening, Early Diagnosis and Management of Severe Combined Immunodeficiency (SCID) in India.
Madkaikar Manisha et al. Indian journal of pediatrics 2016 Feb
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Page last reviewed:
Feb 1, 2024
Page last updated:
May 03, 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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