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) |
|---|---|
| Pages (from-to) | 1266-1267 |
| Number of pages | 2 |
| Journal | Blood |
| Volume | 131 |
| Issue number | 12 |
| DOIs |
|
| State | Published - Mar 22 2018 |
Fingerprint
ASJC Scopus subject areas
- Biochemistry
- Immunology
- Hematology
- Cell Biology
Cite this
Woody eyes, be gone! / Wong, Trisha.
In: Blood, Vol. 131, No. 12, 22.03.2018, p. 1266-1267.Research output: Contribution to journal › Comment/debate
}
TY - JOUR
T1 - Woody eyes, be gone!
AU - Wong, Trisha
PY - 2018/3/22
Y1 - 2018/3/22
N2 - 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
AB - 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
UR - http://www.scopus.com/inward/record.url?scp=85047612185&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85047612185&partnerID=8YFLogxK
U2 - 10.1182/blood-2018-01-828194
DO - 10.1182/blood-2018-01-828194
M3 - Comment/debate
C2 - 29567753
AN - SCOPUS:85047612185
VL - 131
SP - 1266
EP - 1267
JO - Blood
JF - Blood
SN - 0006-4971
IS - 12
ER -