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Mol Med. 2014 Dec 16;20 Suppl 1:S37-42. doi: 10.2119/molmed.2014.00187.

Advances in sickle cell therapies in the hydroxyurea era.

Author information

1
Medical Sciences Institute, BloodCenter of Wisconsin, Milwaukee, Wisconsin, United States of America Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.
2
Dana-Farber Cancer Institute, Boston, Massachusetts, United States of America Boston Children's Hospital, Boston, Massachusetts, United States of America Harvard Medical School, Boston, Massachusetts, United States of America.

Abstract

In the hydroxyurea era, insights into mechanisms downstream of erythrocyte sickling have led to new therapeutic approaches for patients with sickle cell disease (SCD). Therapies have been developed that target vascular adhesion, inflammation and hemolysis, including innovative biologics directed against P-selectin and invariant natural killer T cells. Advances in hematopoietic stem cell transplant and gene therapy may also provide more opportunities for cures in the near future. Several clinical studies are underway to determine the safety and efficacy of these new treatments. Novel approaches to treat SCD are desperately needed, since current therapies are limited and rates of morbidity and mortality remain high.

PMID:
25549232
PMCID:
PMC4374518
DOI:
10.2119/molmed.2014.00187
[Indexed for MEDLINE]
Free PMC Article

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