Post-Transcriptional Genetic Silencing ofBCL11Ato Treat Sickle Cell Disease | Zendy

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

AI Assistant Blog Pricing

Home ZAIA Blog

Open Access

Post-Transcriptional Genetic Silencing ofBCL11Ato Treat Sickle Cell Disease

Author(s) -

Erica B. Esrick,

Leslie E. Lehmann,

Alessandra Biffi,

Maureen Achebe,

Christian Brendel,

Marioara F. Ciuculescu,

Heather Daley,

Brenda L. MacKin,

Emily A. Morris,

Amy Federico,

Daniela Abriss,

Kari Boardman,

Radia Khelladi,

Kit L. Shaw,

Hélène Negre,

Olivier Nègre,

Sarah Nikiforow,

Jerome Ritz,

SungYun Pai,

Wendy B. London,

Colleen Dansereau,

Matthew M. Heeney,

Myriam Armant,

John P. Manis,

David A. Williams

Publication year - 2020

Publication title -

new england journal of medicine

Language(s) - Uncategorized

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa2029392

Subject(s) - gene silencing , disease , cell , genetics , medicine , biology , gene

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 is a repressor of γ-globin expression and HbF production in adult erythrocytes. Its down-regulation is a promising therapeutic strategy for induction of HbF.

The content you want is available to Zendy users.

Already have an account? Click here to sign in.

Having issues? You can contact us here

Accelerating Research