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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Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Article
Acesso em linha: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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