Novel Sickle Cell Disease Therapies: Targeting Pathways Downstream of Sickling

Kerry A. Morrone, William Beau Mitchell, Deepa G. Manwani

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Sickle cell disease is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. Although the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso-occlusion results from heterotypic interactions between the altered, adhesive sickle cell RBCs, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These downstream targets are the focus of emerging treatments with considerable potential to ameliorate disease manifestations. This review summarizes the progress on development of these agents.

Original languageEnglish (US)
JournalSeminars in Hematology
DOIs
StateAccepted/In press - Jan 1 2018

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Abnormal Hemoglobins
Hemoglobinopathies
Hemolytic Anemia
Sickle Cell Anemia
Hemostatics
Cell- and Tissue-Based Therapy
Hemolysis
Reperfusion Injury
Oxidants
Polymerization
Adhesives
Endothelium
Neutrophils
Blood Platelets
Quality of Life
Inflammation
Therapeutics

Keywords

  • Adhesion pathway
  • Inflammation
  • Novel agents
  • Sickle cell disease

ASJC Scopus subject areas

  • Hematology

Cite this

Novel Sickle Cell Disease Therapies : Targeting Pathways Downstream of Sickling. / Morrone, Kerry A.; Mitchell, William Beau; Manwani, Deepa G.

In: Seminars in Hematology, 01.01.2018.

Research output: Contribution to journalArticle

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