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Public Health Genomics and Precision Health Knowledge Base (v9.0)
PHGKB
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Public Health Genomics Branch
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Precision Health Database
Precision Health Database|Search|Public Health Genomics and Precision Health Knowledge Base (PHGKB)
Last data update: May 03, 2024
. (Total: 63769 Documents since 2012)
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The FDA and Gene Therapy for Duchenne Muscular Dystrophy
Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial.
Jinyuan Wang et al. Signal Transduct Target Ther 2024 9(1) 95
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A multinational survey of potential participant perspectives on ocular gene therapy.
Alexis Ceecee Britten-Jones et al. Gene Ther 2024
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A scoping review exploring cure definitions and language for inherited hemoglobinopathies.
Marilyn S Baffoe-Bonnie et al. Genet Med Open 2024 2
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A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies.
Sumaya Abuloha et al. Hum Gene Ther 2024
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Twenty years of Gendicine® rAd-p53 cancer gene therapy: The first-in-class human cancer gene therapy in the era of personalized oncology.
Li Qi et al. Genes Dis 2024 11(4) 101155
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An International Learning Collaborative Phase 2 Trial for Haploidentical Bone Marrow Transplant in Sickle Cell Disease.
Adetola A Kassim et al. Blood 2024
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Current state of gene therapy in sickle cell disease.
Mei San Tang et al. Vox Sang 2024
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Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment.
Teresa H Y Wu et al. Mol Genet Metab 2024 142(1) 108349
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Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
Michiel Coppens et al. Lancet Haematol 2024
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Patient experiences and perceived value of genetic testing in inherited retinal diseases: a cross-sectional survey.
Alexis Ceecee Britten-Jones et al. Sci Rep 2024 14(1) 5403
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An update review of new therapies in sickle cell disease: the prospects for drug combinations.
Sanne Lugthart et al. Expert Opin Pharmacother 2024
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Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study.
Simon Fletcher et al. Orphanet J Rare Dis 2024 19(1) 59
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Blood Spotlight Review on Gene Therapy for Sickle Cell Disease.
Edward J Benz et al. Blood 2024
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Redefining informed consent form in cell and gene therapy trials.
Varsha Dalal et al. Perspect Clin Res 2024 15(1) 4-9
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Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.
Anirban Basu et al. Ann Intern Med 2024
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Building CRISPR Gene Therapies for the Central Nervous System: A Review.
Sally E Salomonsson et al. JAMA Neurol 2024
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Deaf boy can now hear after breakthrough gene treatment
Medical XPress, January 23, 204
Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders.
Marlen C Lauffer et al. Commun Med (Lond) 2024 4(1) 6
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KNOWLEDGE AND ATTITUDES TOWARDS GENE THERAPY OF A COHORT OF ITALIAN PATIENTS WITH HAEMOPHILIA.
Ilaria Cutica et al. J Thromb Haemost 2023
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Perspectives and perception of haemophilia gene therapy by French patients.
Geneviève Pietu et al. Haemophilia 2023
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An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States.
Austin Wesevich et al. Ann Intern Med 2023
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Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa.
Nchangwi Syntia Munung et al. Gene Ther 2023
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Genome editing for sickle cell disease: still time to correct?
Giulia Ceglie et al. Front Pediatr 2023 111249275
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The Lived Experience of Pediatric Gene Therapy - A Scoping Review.
Laura Kimberly et al. Hum Gene Ther 2023
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Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid.
Antal Zemplenyi et al. Pharmacoeconomics 2023
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Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK.
Sara Boyce et al. Orphanet J Rare Dis 2023 18(1) 366
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A new age of precision gene therapy.
Axel Schambach et al. Lancet 2023
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Public Health Genetics, Gene Therapy, and Duchenne Muscular Dystrophy
CDC Webinar, December 18, 2023
Gene therapy-based strategies for spinal muscular atrophy-an Asia-Pacific perspective.
Michelle A Farrar et al. Mol Cell Pediatr 2023 10(1) 17
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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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