Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

BACKGROUND: Sickle cell disease is characterized by hemolytic anemia, pain, and progressive organ damage. A high level of erythrocyte fetal hemoglobin (HbF) comprising α- and γ-globins may ameliorate these manifestations by mitigating sickle hemoglobin polymerization and erythrocyte sickling. BCL11A...

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Foilsithe in:N Engl J Med
Main Authors: Esrick, Erica B., Lehmann, Leslie E., Biffi, Alessandra, Achebe, Maureen, Brendel, Christian, Ciuculescu, Marioara F., Daley, Heather, MacKinnon, Brenda, Morris, Emily, Federico, Amy, Abriss, Daniela, Boardman, Kari, Khelladi, Radia, Shaw, Kit, Negre, Helene, Negre, Olivier, Nikiforow, Sarah, Ritz, Jerome, Pai, Sung-Yun, London, Wendy B., Dansereau, Colleen, Heeney, Matthew M., Armant, Myriam, Manis, John P, Williams, David A.
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2020
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Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7962145/
https://ncbi.nlm.nih.gov/pubmed/33283990
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa2029392
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