Abstract
In this issue of Blood, Shapiro et al describe the first-in-class experience of infusing plasma-derived Glu-plasminogen to 14 patients with congenital deficiency of plasminogen as part of an ongoing, phase 2/3, open-label clinical trial, reporting encouraging results. 1 Congenital plasminogen deficiency is caused by homozygous or compound-heterozygous mutations in the plasminogen (PLG) gene, located on chromosome 6q26. It is, in the words of the authors, “ultra-rare,” predicted to affect ∼1 to 2 per million people. 2 Plasminogen is activated to plasmin and is critical for intravascular and extravascular fibrinolysis. Other functions include wound healing, cell migration, tissue remodeling, angiogenesis, and embryogenesis. Glu-plasminogen has a glutamic acid residue at its N-terminus, as opposed to Lys-plasminogen, which has a lysine. Glu-plasminogen is the predominant form found in circulation and has a half-life of 2 to 2.5 days, whereas the half-life of Lys-plasminogen is 0.8 days. 3
Original language | English (US) |
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Pages (from-to) | 1266-1267 |
Number of pages | 2 |
Journal | Blood |
Volume | 131 |
Issue number | 12 |
DOIs |
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State | Published - Mar 22 2018 |
Externally published | Yes |
ASJC Scopus subject areas
- Biochemistry
- Immunology
- Hematology
- Cell Biology