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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Detaylı Bibliyografya
Yayımlandı:N Engl J Med
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2020
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Article
Online Erişim: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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