Of mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease
Author:
Publisher
Elsevier BV
Subject
General Biochemistry, Genetics and Molecular Biology
Reference15 articles.
1. Post-transcriptional genetic silencing of BCL11A to Treat sickle cell disease;Esrick;N. Engl. J. Med.,2021
2. CRISPR-Cas9 gene editing for sickle cell disease and beta-thalassemia;Frangoul;N. Engl. J. Med.,2021
3. Reactivation of a developmentally silenced embryonic globin gene;King;Nat. Commun.,2021
4. Transcription factor competition at the gamma-globin promoters controls hemoglobin switching;Liu;Nat. Genet.,2021
5. Treating rare diseases in Africa: the Drugs exist but the need is Unmet;Luzzatto;Front Pharmacol.,2021
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1. Identification of novel BCL11A variant in a patient with developmental delay and behavioural differences;International Journal of Developmental Neuroscience;2024-08-26
2. CRISPR technology in human diseases;MedComm;2024-07-29
3. A Journey from Blood Cells to Genes and Back;Annual Review of Genomics and Human Genetics;2023-08-25
4. The Italian Association of Epidemiology (AIE) how we started it and my hopes for its future;EPIDEMIOL PREV;2023
5. Precision Medicine and Sickle Cell Disease;HemaSphere;2022-08-18
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