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

Erica B. Esrick,Leslie E. Lehmann,Alessandra Biffi,Maureen Achebe,Christian Brendel,Marioara F. Ciuculescu,Heather Daley,Brenda MacKinnon,Emily Morris,Amy Federico,Daniela Abriss,Kari Boardman,Radia Khelladi,Kit L. Shaw,Helene Negre,Olivier Negre,Sarah Nikiforow,Jerome Ritz,Sung-Yun Pai,Wendy B. London,Colleen Dansereau,Matthew M. Heeney,Myriam Armant,John P. Manis,David A. Williams

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

2020

Abstract 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 ...

Keywords:

organ damage
Immunology
Hemolytic anemia
Cell
Fetal hemoglobin
Disease
Gene silencing
Medicine

Correction
Source
Cite
Save
Machine Reading By IdeaReader

References

Citations