| Autor: |
Akito Nakagawa, Marissa K. Cooper, Maria Kost-Alimova, James Berstler, Binglan Yu, Lorenzo Berra, Elizabeth S. Klings, Mary S. Huang, Matthew M. Heeney, Donald B. Bloch, Warren M. Zapol |
| Rok vydání: |
2022 |
| Předmět: |
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| Zdroj: |
ACS Omega. 7:14009-14016 |
| ISSN: |
2470-1343 |
| DOI: |
10.1021/acsomega.2c00541 |
| Popis: |
Sickle cell disease (SCD) is an inherited disorder of hemoglobin (Hb); approximately 300,000 babies are born worldwide with SCD each year. In SCD, fibers of polymerized sickle Hb (HbS) form in red blood cells (RBCs), which cause RBCs to develop their characteristic "sickled" shape, resulting in hemolytic anemia and numerous vascular complications including vaso-occlusive crises. The development of novel antisickling compounds will provide new therapeutic options for patients with SCD. We developed a high-throughput "sickling assay" that is based on an automated high-content imaging system to quantify the effects of hypoxia on the shape and size of RBCs from HbSS SCD patients (SS RBCs). We used this assay to screen thousands of compounds for their ability to inhibit sickling. In the assay, voxelotor (an FDA-approved medication used to treat SCD) prevented sickling with a |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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